Podcasts about Duchenne muscular dystrophy

Agreement between government and parents of children with Duchenne Muscular Dystrophy, Prime Minister's admission that he is second in command to Ivanishvili, Reactions to Armenian elections, New Strategic Partnership Agreement with China, Arrest of Canadian Student for ADHD medication. Thanks for tuning in!Let us know what you think and what we can improve on by emailing us at info@rorshok.com Like what you hear? Subscribe, share, and tell your buds.Report by Georgian Young Lawyers Association - Personalized Lawmaking in Georgia as a means of unjustly restricting rights: https://admin.gyla.ge/uploads_script/publications/pdf/PERSONALIZED%20LAWMAKING%20IN%20GEORGIA%20AS%20A%20MEANS%20OF%20UNJUSTLY%20RESTRICTING%20RIGHTS.pdf Georgia Disenfranchised by Sergi Kapanadze: https://politicsgeo.com/georgia-disenfranchised/ Check out our new t-shirts: https://rorshok.store/We want to get to know you! Please fill in this mini-survey: https://forms.gle/NV3h5jN13cRDp2r66Wanna avoid ads and help us financially? Follow the link: https://bit.ly/rorshok-donate

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into some of the latest news shaping the industry, from breakthroughs in cancer therapies to advancements in AI-driven drug discovery. Starting with regulatory updates, the potential appointment of Richard Pazdur, M.D., as the new FDA Commissioner is causing quite a stir. Following Marty Makary's resignation, Pazdur has emerged as a prominent candidate due to his extensive background in oncology drug regulation. Known for his commitment to accelerating cancer therapy approvals, his potential leadership could maintain or even amplify the focus on expediting innovative treatments for cancer patients. In a significant regulatory achievement, Beone Medicines celebrated the FDA's approval of Beqalzi, marking it as the first BCL-2 inhibitor approved for mantle cell lymphoma. This approval challenges AbbVie's Venclexta and underscores a growing trend towards targeted cancer therapies that offer new treatment avenues for patients. The oncology space continues to be fiercely competitive, with companies striving to deliver more precise and effective cancer treatments. Turning to clinical trials, AstraZeneca's Imfinzi has shown promising results in a phase 3 trial focused on bladder cancer patients who are not eligible for cisplatin-based chemotherapy. These findings position Imfinzi as a strong competitor to Merck's Keytruda and reinforce AstraZeneca's strategic focus on expanding its oncology portfolio through novel combinations and indications. In the realm of genetic therapies, Regenxbio has achieved a milestone with its gene therapy for Duchenne muscular dystrophy. This therapy met its primary endpoint in pivotal trials, highlighting the potential of gene therapies to address rare diseases with limited treatment options. Such successes are likely to encourage further investment in gene editing technologies, which hold significant promise for tackling conditions once deemed untreatable. The FDA is also exploring frameworks to repurpose existing drugs for new uses by leveraging existing safety data. This could streamline drug development processes and offer cost-effective solutions for patients with complex conditions. However, this approach will need rigorous validation of efficacy in new indications to ensure patient safety and therapeutic effectiveness. Despite setbacks in its Alzheimer's research, Biogen remains steadfast in its efforts. While their tau-targeting candidate did not meet primary endpoints in a phase 2 trial, reductions in tau pathology and cognitive benefits were observed. This perseverance showcases Biogen's commitment to finding innovative approaches to tackle Alzheimer's disease despite ongoing challenges. On the operational front, Taiwan's Bora Group is acquiring Macrogenics' CDMO operations for up to $127.5 million. This move reflects a broader trend of consolidation within the CDMO space as companies aim to enhance their production capabilities and streamline operations. Quality control remains a critical concern as evidenced by Sun Pharma's recent recall of a chemotherapy batch due to glass particle contamination. Incidents like these underline the importance of stringent quality assurance measures throughout the manufacturing process to ensure patient safety. Moreover, Viz.ai has launched an AI-powered pulmonary care platform aimed at integrating acute and chronic care workflows. This development signals an increasing adoption of artificial intelligence in healthcare, promising improvements in diagnostics and patient management efficiency. AI continues to gain traction as Isomorphic Labs recently secured $2.1 billion in Series B funding aimed at enhancing AI-driven drug design models. Similarly, Charles River has introduced an AI-powered digital pathology platform poised to Support the show

In this episode, Morah Tziri speaks with two truly InKredible Kids.Chaim is a 12-year-old Team Lifeline runner who has already completed the Miami Half Marathon three times. He's funny, full of energy, and has a message for every kid listening: no one is too young to make a difference.Then, in a surprise twist, Chaim gets to reconnect with Azzi, a boy he met at the marathon, who inspires him deeply. Azzi shares about music, Camp Simcha, life in a wheelchair, and how he faces challenges with simcha and emunah.One runs. One sings. Both keep going.Watch the music video we talked about: In this episode, Morah Tziri talks about watching Azzi's powerful “Titanium” music video from Camp Simcha. After you listen, make sure to watch the video too. It gives you a whole new appreciation for Azzi's strength, simcha, and talent.Watch here:Azzi's Camp Simcha Titanium Video Thank you to our sponsor, WheePlay by SR. Check them out at wheesr.com.New InKredible Kids x TYH Nation coloring sheets are available every Thursday at tyhnation.com.For a whole world of resources for your kids, head to inkrediblekids.org now. Be sure to sign up for our email so you never miss new content and live programs.For sponsorships, ads, collaborations, or  bookings: info@inkrediblekids.orgAnd remember: You are all inkredible kids.Support the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. In a rapidly evolving industry landscape, artificial intelligence has emerged as a game-changer, transforming how major pharmaceutical companies approach drug development and diagnostics. Roche's strategic acquisition of PathAI for over $1 billion exemplifies this shift, highlighting the growing importance of AI-driven diagnostics in digital pathology. This move signals a broader trend where AI is no longer just a theoretical concept but a practical tool enhancing healthcare delivery. Alnylam's recent challenges with its web-based presentation of Amvuttra data underscore the need for accuracy in representing clinical data digitally—showing that while AI can offer innovative ways to present data, it also demands rigorous standards to ensure clarity and prevent misleading claims. On the regulatory front, the FDA's evolving stance on cell therapy Ebvallo, alongside its new one-day assessment pilot program, is shaping the industry's regulatory environment. The reconsideration of Ebvallo's earlier rejection due to single-arm trial data concerns illustrates the FDA's willingness to adapt its regulatory framework to accommodate innovative therapies. This adjustment could potentially pave the way for other gene therapies in development, including those by UniQure. Moreover, Sanofi's withdrawal of Tzield from the FDA's contentious Commissioner's National Priority Voucher program reflects ongoing debates about expedited review processes, underscoring the need for transparent and efficient pathways for bringing new therapies to patients swiftly. Additionally, the FDA's AI-guided inspection pilot aims to modernize compliance strategies and enhance pharmaceutical manufacturing oversight. In clinical trials, companies continue to face both triumphs and setbacks. Entrada Therapeutics experienced a significant decline in stock value following underwhelming Duchenne Muscular Dystrophy trial results, potentially reshaping competitive dynamics in favor of rivals like Novartis. Conversely, Angelini Pharma is making strategic moves with its $4.1 billion acquisition of Catalyst Pharmaceuticals, targeting market expansion in the U.S. through Firdapse, which is poised to make significant impacts in treating rare diseases. MingMed Biotechnology's promising phase 2 results for QA102 could signal new treatment paradigms for dry AMD—a condition with limited current interventions. Therapeutic innovation is also being driven by policy shifts that encourage research into psychedelic drugs for mental health treatment. Optimi Health's IPO indicates growing investor interest in this area, fueled by regulatory easing under recent executive orders aimed at facilitating psychedelic research. Strategic pipeline adjustments are evident as companies realign their focus based on emerging data insights. Ascendis Pharma's decision to halt its IL-2 oncology program marks a shift toward more promising avenues, while Beone Medicines' restructuring reflects a similar strategy by discontinuing several early-stage cancer programs. The industry's dynamism is further illustrated by Eli Lilly's substantial $4.5 billion investment in expanding its Indiana campus. This move not only enhances Lilly's capacity for genetic medicine and metabolic disease manufacturing but also underscores a broader industry commitment to precision medicine and biologics—fields anticipated to play pivotal roles in future healthcare solutions. Meanwhile, Bayer's acquisition of Perfuse Therapeutics seeks to bolster its ophthalmology portfolio, addressing significant unmet needs in eye disease treatments. Novo Nordisk's success with Wegovy highlights strong market demand for effective obesity treatments, demonstrating an industry-wide shift towards addressing lifestyle-related diseases. LegislSupport the show

In this episode of The Grief Lounge, I'm joined by Jack Waddington. Jack is an author, having written his first book after the loss of his brother Sam. I'm on a journey to see you Sam . . Jack is also an artist, who illustrated the cover for the book, and a secondary school teacher. In this episode Jack and I have a deeply honest conversation about sibling grief, loss and the lifelong bond between brothers.Jack shares the story of his brother Sam, who lived with Duchenne muscular dystrophy and died at the age of 26.We talk about what it was like growing up alongside Sam, the role Jack played as a sibling and carer, and how Duchenne shaped not just Sam's life, but the whole family's.This is a conversation about love, responsibility and the kind of connection that does not end with death.Jack speaks openly about the shock of losing his brother, the guilt that can come with sibling loss, and the reality of navigating grief as a young man.We also talk about men's mental health and the importance of creating space for honest conversations around grief, vulnerability and emotion.Writing became a way for Jack to process his loss and stay connected to Sam. His book, I'm on a Journey to See You Sam, is both a tribute to his brother and a way of raising awareness about Duchenne muscular dystrophy, disability and the experience of sibling grief.If you have experienced the loss of a sibling, are supporting someone who has, or want to better understand the impact of Duchenne muscular dystrophy, this conversation offers honesty, insight and connection.More about Jack.Jack is a London-based writer, practising artist, and art teacher. His debut memoir, I'm on a Journey to See You, Sam, began as diary entries written in the days and months after the death of his younger brother, who lived with Duchenne muscular dystrophy. He was also selected for the Local Author Showcase at the 2026 Ealing Book Festival.Alongside writing, Jack creates drawings and paintings, and designed the book's cover artwork himself. He lives in London with his wife. Jack continues to write, teach art, and explore ways to work with keeping his relationship with Sam very much connected. You can follow and connect with Jack on Instagram @jackwaddington_ You can purchase I'm on a journey to see you Sam from Amazon here https://amzn.eu/d/0eadhISAIf you would like to join our Facebook Community page where you will be joined by other navigating grief here is the linkhttps://www.facebook.com/groups/thegrieflounge/My website is www.thegriefloungeuk.com and my instagram account is @the_grief_coach_uk Thank you so much for listening to this episode. Please share the link for the podcast to anyone you may feel would benefit.You do not have to navigate the path of grief alone.

Dr. Megan Waldrop visits the studio as we consider Duchenne Muscular Dystrophy. This genetic condition remains a serious diagnosis, but there is also real progress happening in how we diagnose, treat, and support those living with this condition. Tune in for details!

Around 30% of boys diagnosed with Duchenne muscular dystrophy also experience cognitive dysfunction and neurodevelopmental disorders like autism and ADHD. A UT Health San Antonio neuroscientist is doing research he hopes will uncover what is causing these deficits and how they might be treated.

A conversation with Carol RoopchandsinghHe never stopped fighting. Neither will she.Matthew Roopchandsingh - known to many as Lil Roopie - lived for nearly thirty years with Duchenne Muscular Dystrophy. He built a YouTube channel, designed his own logo, and spent his life reaching out to others with disabilities, telling them never to give up hope. He passed away on November 14th, 2024.His mother, Carol Roopchandsingh, was his caregiver, his advocate, and his guardian. She worked three jobs. She slept in hospital chairs. She fought insurance companies, dismissive doctors, and a system that too often asked: why provide care to someone who will never get better?Today, on our milestone 100th episode, Carol bravely sat with Dr. Fethke to bring Matthew's story out of the shadows - and to make sure his legacy is just beginning.I am honored to introduce Carol Roopchandsingh.—We spoke about the long and painful road to Matthew's Duchenne diagnosis, the systemic failures that denied him the equipment, therapy, and support he needed, the emotional and physical toll of caregiving with no safety net, how Matthew channelled his condition into a platform of advocacy and joy, and Carol's vision for a foundation in his memory - to fight for every person who lives with disability, that the system forgets.Lil Roopie's YouTube Channel: https://www.youtube.com/@lilroopie/featuredFollow me on Instagram and Facebook @ericfethkemd and checkout my website at www.EricFethkeMD.com. My brand new book, The Privilege of Caring, is out now on Amazon! https://www.amazon.com/dp/B0CP6H6QN4

Has the FDA thwarted Donald Trump on his Right to Try initiative in 2018? Angelina Olivera tells me about her son Ryu, who suffers from a rare disease, Duchenne Muscular Dystrophy. Ryu is running out of time for an effective medical treatment, but the FDA is blocking access to one drug that shows great promise. Angelina talks to us about fighting the bureaucracy at the FDA, and the need to have promises kept now rather than somewhere down the road.

Has the FDA thwarted Donald Trump on his Right to Try initiative in 2018? Angelina Olivera tells me about her son Ryu, who suffers from a rare disease, Duchenne Muscular Dystrophy. Ryu is running out of time for an effective medical treatment, but the FDA is blocking access to one drug that shows great promise. Angelina talks to us about fighting the bureaucracy at the FDA, and the need to have promises kept now rather than somewhere down the road.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a range of topics that illustrate the rapidly changing landscape of our industry. We'll discuss everything from vaccine expansions to regulatory challenges, highlighting both the breakthroughs and hurdles faced by companies worldwide.Kicking off with GSK's recent achievement, their respiratory syncytial virus (RSV) vaccine, Arexvy, has gained FDA approval for adults aged 18 to 49 at increased risk of lower respiratory tract disease. This marks a significant milestone not only for GSK but also in the competitive RSV vaccine market where Pfizer and Moderna have already made their mark. This development underscores the industry's ongoing commitment to preventing RSV-related complications, reflecting the fierce competition driving innovation in vaccine development.In parallel, Gilead Sciences is making strides with its HIV pre-exposure prophylaxis medication, Yetztugo. The emphasis here is on how early production strategies can set a medication up for success by ensuring accessibility. This focus on manufacturing highlights a broader trend where efficient production methods are key to delivering timely healthcare solutions, underscoring the integral role manufacturing plays in modern drug development.Legend Biotech is navigating the competitive dynamics of the CAR-T therapy market with its treatment, Carvykti, despite facing competition from Johnson & Johnson's bispecific antibody Tecvayli. This scenario highlights strategic partnerships and market positioning challenges within oncology treatments, illuminating the complex landscape companies must navigate to succeed.On the regulatory front, Hyloris Pharmaceuticals encountered a setback when the FDA issued a Complete Response Letter due to manufacturing issues with its antiviral valacyclovir oral suspension. This incident serves as a reminder of the stringent manufacturing standards regulatory bodies demand and the essential nature of compliance in successful drug development.In an exciting advancement out of China, Neuracle Technology has developed the country's first brain-computer interface implant for paralyzed patients. This pioneering neurotechnology involves implanted EEG electrodes connected to a robotic glove capable of grasping objects. Such innovations highlight China's increasing role in cutting-edge medical technology development and represent a significant leap forward in rehabilitation for patients with severe disabilities.However, not all news is positive. Immutep faced an unexpected phase 3 failure with its LAG-3 candidate, which surprised analysts and led to a stock decline. These outcomes underscore the inherent risks in drug development and stress the importance of rigorous clinical evaluation to ensure both efficacy and safety.Shifting focus to diabetes management, companies like Insulet, Abbott, and Dexcom are expanding their efforts toward personalized care through continuous glucose monitors (CGMs) and insulin pumps. These advancements are part of an ongoing trend toward personalized diabetes management tools aimed at improving glucose control for patients.Meanwhile, Simtra Biopharma Solutions received an FDA warning due to contamination issues at one of its production facilities. This serves as a stark reminder of how crucial it is to maintain high-quality standards in drug manufacturing processes to avoid disruptions and ensure patient safety.Strategically speaking, we see notable shifts as companies like Eli Lilly invest in Asia and Pfizer enters obesity treatment markets through strategic partnerships. These moves highlight globalization trends as pharma companies seek to address high unmet medical needs in emerging markets.In a significant transition for BioNTech, founders Ugur Sahin and Ozlem Tureci are preparing to leave Support the show

In this episode, Dan Murphy, Executive Director and Founder of Decker's Blueprint, shares the deeply personal journey that began when his son Decker was diagnosed with Duchenne Muscular Dystrophy in 2024. Faced with a devastating prognosis, Dan refused to accept “wait and see” as an option. Instead, he applied a builder's mindset to the fight—launching Decker's Blueprint to fund promising scientific research, accelerate access to treatments, and challenge the traditional pace of progress.Dan opens up about the urgency behind his mission, why he believes time is muscle, and how families, researchers, and advocates can work together to turn a once-fatal diagnosis into a manageable disease. This powerful conversation is a testament to resilience, action, and the relentless pursuit of hope in the face of impossible odds.???? Connect with Dan Murphy: ✅ Website: https://www.deckersblueprint.org/ ✅ Facebook: https://www.facebook.com/deckers.blueprint/ ✅ Instagram: https://www.instagram.com/deckersblueprint/?hl=en ✅ LinkedIn: https://www.linkedin.com/company/decker-s-blueprint/ Thank you for watching Lancaster Connects! This is the show about small business and small charity success in Lancaster county - we showcase the battle on Main Street, big vs. small David vs Goliath, and bring you the best of what makes Lancaster so great. ???? Want to create live streams like this? Check out StreamYard: https://StreamYard.CastAhead.net ➡️ Get your FREE copy of Ben McClure and Jeff Giagnocavo's book - "Sleep Better" https://gardnersmattressandmore.com/sleep-betterLIVE SHOW PODCAST & REPLAYS: ???? Connect with Lancaster Connects:✅ Official: https://lancasterconnects.com/ ✅ YouTube: https://www.youtube.com/@LancasterConnects ✅ LinkedIn: https://www.linkedin.com/company/lancaster-connects✅ Facebook: https://www.facebook.com/LancasterConnectsLancaster Connects is produced by Chris Stone at Cast Ahead:  https://CastAhead.net #LancasterCounty #LancasterPA #LancasterCountyPA #LancasterConnects #Community #RetailExperience

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into some pivotal advancements and strategic shifts within the industry, highlighting how these changes are shaping the future of patient care and drug development.Let's start with Bristol Myers Squibb, which has been making headlines with its latest success in the realm of antibody-drug conjugates (ADCs). The company's ADC has reached an important milestone in a Phase 3 breast cancer trial conducted in China. This study successfully met its dual primary survival endpoints, affirming the company's significant $800 million investment in this promising drug candidate. The potential of ADCs in oncology cannot be overstated; they offer a remarkable combination of targeted therapy by harnessing the specificity of antibodies alongside the cytotoxic power of traditional chemotherapy. This approach not only enhances precision in treatment but also minimizes collateral damage to healthy tissues, showcasing the transformative potential of ADCs in cancer therapy.On the regulatory front, there are ongoing discussions about the impact of political decisions on drug pricing and innovation. The Trump administration's Most Favored Nation drug pricing policy has stirred significant concern within the biotech sector. In response, ten midsize biotech firms have united to form the Midsized Biotech Alliance of America to challenge this policy. They argue that such pricing strategies could hinder innovation by enforcing restrictive pricing models, potentially stalling the development pipeline for new therapies that address unmet medical needs.In terms of strategic corporate movements, Boehringer Ingelheim has entered into a $500 million partnership with a British biotech firm aimed at developing an oral therapy for autoimmune diseases. This collaboration is part of a broader trend towards precision medicine which focuses on modulating specific immune cells to improve treatment outcomes while minimizing unwanted side effects. It's a clear indication that companies are increasingly investing in targeted therapies that promise better efficacy and patient safety. Additionally, Boehringer Ingelheim's partnership with Sitryx underscores another trend: strategic partnerships aimed at innovative research endeavors with substantial investment commitments—potentially exceeding $500 million—to explore immune response modulation.The acquisition landscape is also seeing dynamic shifts. Asahi Kasei's acquisition of Germany's AiCuris for $920 million marks a strategic move to enhance its R&D capabilities, specifically focusing on antiviral therapies for immunocompromised patients. This acquisition aligns with growing global attention towards infectious disease research, especially in a post-pandemic era where preparedness and rapid response capabilities have become paramount.Meanwhile, Sarepta Therapeutics is undergoing a significant leadership change as CEO Doug Ingram announces his retirement. Ingram's leadership was characterized by notable advancements in treatments for Duchenne muscular dystrophy (DMD), although it wasn't without its share of challenges regarding regulatory and pricing debates. As Sarepta continues to expand its gene therapy pipeline, this leadership transition comes at a crucial juncture, potentially setting new directions for the company's future.Accent Therapeutics' recent decision to halt its solid tumor trial due to adverse events exemplifies the risks inherent in drug development. The company is now redirecting its focus towards other cancer programs, illustrating how adaptability remains key in navigating clinical setbacks.Protagonist Therapeutics has made a strategic choice by accepting a $400 million payment from Takeda instead of sharing profits from its hematology asset rusfertide. This decision may provideSupport the show

Jeff Braverman joins HarmonyTALK to reflect on 30 years of leadership at Camp Nesher and the unexpected decisions that helped shape one of its most meaningful legacies.What began as a dream to create a vibrant summer camp experience centered on community and faith took an unexpected turn when he was approached by a parent who asked if their wheelchair-bound child with Duchenne Muscular Dystrophy could be accommodated and welcomed at camp. Braverman’s decision to move forward would become one of the most defining moments of his career. He didn’t simply advocate for accessibility. He embedded it into the culture of the camp itself, shaping programs and practices that welcomed campers of all abilities as equal participants through a partnership program with the national organization Yachad (Hebrew for “together”,) whose mission is inclusion for individuals with developmental disabilities into the greater Jewish community. Located in Lakewood, Pennsylvania, Camp Nesher is part of NJY Camps, a 105-year-old organization and one of the world’s largest camping agencies, awarding up to $2 million in scholarships each year. In addition, NJY brings more than 40 years of experience in inclusive programming through its Round Lake inclusion program at its Milford, Pennsylvania, property which includes Camps Nah-Jee-Wah, Cedar Lake and Teen Camp. In this inspiring conversation with host Todd Stephens, Braverman shares leadership lessons learned along the way and how passion, purpose and music can move in harmony. FOLLOW HARMONYTALK PODCAST @harmonytalkpodcast Join Our Mailing List: https://www.harmonytalkpodcast.com/signup Instagram: https://instagram.com/harmonytalkpodcast Facebook: https://www.facebook.com/harmonytalkpodcast YouTube: https://youtube.com/@HarmonyTALKPodcast LinkedIn: https://www.linkedin.com/company/harmonytalkpodcast https://harmonytalkpodcast.com/ Follow Host, Todd Stephens: https://www.instagram.com/workinman1/ Email harmonytalkpodcast@gmail.com for sponsorship and guest opportunities! See omnystudio.com/listener for privacy information.

When Joshua Ruff's heart stopped for three minutes, everything changed.Living with Duchenne Muscular Dystrophy (DMD) since childhood, Joshua had already navigated a lifetime of disability, adaptation, and resilience. But in 2020, a sudden cardiac arrest during the early days of COVID forced him into a profound reckoning with mortality, fear, and what actually matters.Unable to speak and communicating only through his eyes, Joshua was told he might never return home. Instead, that moment became the catalyst for a new way of living. One centred on human connection, creative purpose, and letting go of fear.In this powerful conversation, Joshua shares how surviving cardiac arrest reshaped his outlook on life, relationships, and ambition. He opens up about growing up with DMD, the emotional toll of teenage years, and the quiet pressure to always appear positive as a wheelchair user. We explore how gardening became both therapy and vocation, leading to the creation of Henle Gardens, a lavender farm producing oil, products, and community experiences.This episode is about disability, yes. But more than that, it is about meaning, independence, love, and choosing to live fully without apology.Key Topics CoveredSurviving a cardiac arrest and communicating only through eye movementLiving with Duchenne Muscular Dystrophy and challenging early life expectancy narrativesLetting go of fear after facing deathGardening as purpose, therapy, and businessBuilding an accessible lavender farm and producing lavender oilIndependence, support systems, and redefining successWhy people with disability are elite problem solversRelationships, self-worth, and rejecting the idea of being a burdenPositivity, grief, and the danger of masking emotionsNotable Moments“The most important thing is human connection. Everything else doesn't matter.”“My heart stopped for three minutes, and somehow that freed me.”“I didn't believe I deserved a relationship. That belief almost cost me one.”“People with disability are the best problem solvers because life never gives us the easy path.”“Independence for me is choice, not doing everything alone.”About Joshua RuffJoshua Ruff is a gardener, lavender producer, and founder of Henle Gardens in regional Victoria. Living with Duchenne Muscular Dystrophy, Joshua has transformed personal adversity into creative expression, community connection, and entrepreneurship.After surviving cardiac arrest in 2020, he committed to building a life driven by purpose rather than fear. Today, his lavender farm produces oil, dried lavender products, and hosts garden visits, festivals, and community groups, proving that accessibility and beauty are not mutually exclusive.

Matt Washington talks about growing up in Washington and building a life in Laguna Beach. He reflects on early jobs, the impact of having family members with Duchenne Muscular Dystrophy, and how martial arts—especially Jiu Jitsu —shaped his discipline and mindset. Matt also breaks down his work as a land man, offering a behind-the-scenes look at the business of buying and selling rural land, the challenges of the industry, and what fuels his ambitions moving forward.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a series of transformative events reshaping the industry landscape, from regulatory advancements to scientific breakthroughs and strategic business maneuvers.Kicking off with a significant regulatory update, the FDA's Rare Pediatric Disease Voucher Program has been rejuvenated through a newly signed government funding bill. This initiative is designed to expedite the development of treatments for rare pediatric diseases, offering crucial incentives to companies targeting this critical healthcare segment. By reauthorizing this program, there's an expectation of stimulating innovation and potentially bringing more treatments to market for conditions with limited existing therapies. This move underscores a broader commitment to addressing unmet medical needs through incentivized innovation.Turning to corporate developments, Eli Lilly is anticipating substantial growth in revenue despite facing pricing pressures on its key products, Mounjaro and Zepbound. The company projects revenues between $80 billion and $83 billion for 2026, marking a 25% increase from 2025 at the midpoint. This growth is attributed to strong product performance and strategic maneuvers within their pipeline. Eli Lilly has also made strategic decisions by optimizing its pipeline through dropping three clinical-stage drugs, including a gene therapy acquired via Prevail Therapeutics. This move points towards Lilly's focus on concentrating efforts on more promising candidates within their expansive pipeline. Additionally, Eli Lilly is expanding its GLP-1 franchise beyond metabolic diseases into immunology and inflammation with ongoing clinical trials in conditions such as asthma, psoriatic arthritis, Crohn's disease, and ulcerative colitis. This strategic expansion could lead to novel therapeutic options for chronic inflammatory diseases.Similarly, Bristol Myers Squibb is focusing on new growth drivers amid declining sales of legacy drugs. With $48.2 billion in revenue projected for 2025 largely stemming from newer products, BMS is strategically repositioning itself to maintain momentum amidst market changes.Novartis faces its largest patent expiry challenge but remains optimistic about its trajectory. CEO Vas Narasimhan suggests robust strategies are in place to counteract these patent expiries, indicating a strong focus on innovation and strategic planning to navigate these hurdles. Novartis is also refining its oncology strategy by cutting early-stage cancer candidates while adding new ones focused on promising therapeutics—a broader trend of adopting data-driven approaches to streamline drug development pipelines.Meanwhile, AbbVie continues its stronghold in the inflammatory bowel disease market with its blockbuster immunology drugs Skyrizi and Rinvoq. These products significantly contribute to AbbVie's $61.1 billion revenue, highlighting their commitment to maintaining leadership in immunology despite competitive pressures from rivals like Johnson & Johnson.Astellas has exceeded expectations with its cancer drug Vyloy overcoming a trial setback to quadruple sales in the third quarter fiscal year 2025 results. This success underscores the resilience and potential of innovative oncology treatments even when faced with clinical challenges.In financial markets, Veradermics successfully raised $256 million through its IPO, signaling strong investor interest in biotech firms with promising dermatological applications. Concurrently, Eikon Therapeutics marked the largest biotech IPO since 2024 with a $381 million listing on Nasdaq, reflecting renewed investor confidence in biotech ventures. Industry trends indicate a resurgence of interest in public markets exemplified by Eikon Therapeutics' upsized IPO alongside Veradermics' successful Support the show

Michael, a North Cork father, shares the heartbreaking reality of caring for his 12-year-old son with Duchenne Muscular Dystrophy in a council home that traps him. Hosted on Acast. See acast.com/privacy for more information.

Welcome to Season 2 of the Orthobullets Podcast.In this episode, we review the high-yield topic of⁠⁠⁠ Duchenne Muscular Dystrophy⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠from the Pediatrics section.⁠Follow ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Orthobullets⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ on Social Media:⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Facebook⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Instagram⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Twitter⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠LinkedIn⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠YouTube

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we explore a series of groundbreaking advancements and strategic collaborations that promise to transform drug development and patient care.In the autoimmune space, Boehringer Ingelheim has made a significant move by securing a $570 million agreement with CDR-Life. This deal centers on a trispecific antibody, a novel therapeutic approach that targets multiple pathways simultaneously, potentially revolutionizing treatments for autoimmune diseases. Boehringer's commitment to these cutting-edge modalities highlights their strategy to leverage novel technologies for more effective therapeutic solutions.Similarly, Celltrion has entered a $744 million collaboration with Kaigene, focusing on two preclinical autoimmune drugs. This partnership marks Celltrion's strategic shift from biosimilars to novel biologics, positioning the company at the forefront of biologic therapeutics. By investing in early-stage research, Celltrion aims to introduce transformative therapies for autoimmune conditions, showcasing the industry's willingness to bet on groundbreaking scientific advancements.In gene editing, Azalea Therapeutics is gaining attention with its focus on permanent genome editing using a dual-vector approach. Backed by $82 million in funding and support from CRISPR pioneer Jennifer Doudna, Azalea is poised to develop potentially curative solutions through single-dose treatments. The credibility lent by a Nobel laureate adds anticipation to their research outcomes, with the potential to significantly impact gene therapy.Shifting focus to clinical trials, Sarepta Therapeutics faces challenges after missing the primary endpoint in its confirmatory trial for Duchenne muscular dystrophy drugs. Despite this setback, Sarepta is pursuing full FDA approval, emphasizing the complex interplay between clinical data and regulatory strategies. This situation underscores the critical importance of robust confirmatory trials in securing drug approvals and ensuring patient access to new therapies.Merck is making strategic moves in oncology by regaining full control over an early-phase asset and securing $700 million from Blackstone for its oncology pipeline. This dual focus on asset acquisition and financial fortification reflects Merck's aggressive growth strategy aimed at expanding its cancer treatment offerings.Emerging from stealth mode, Neok Bio has secured a $75 million investment to advance bispecific antibody-drug conjugates into clinical trials. These bispecific ADCs represent the forefront of targeted cancer therapies, aiming for precision targeting of cancer cells while minimizing off-target effects. Neok Bio's progress could significantly enhance oncology treatment paradigms through improved therapeutic indices.Turning to regulatory landscapes, Teva's recall of over half-a-million bottles of prazosin hydrochloride due to potential carcinogenic impurities highlights ongoing challenges in ensuring drug safety and quality control within manufacturing processes. Such recalls underscore the critical importance of maintaining high standards in pharmaceutical production.In broader industry developments, we see dynamic trends where scientific innovation meets strategic business decisions and regulatory considerations. The potential impact on patient care is profound, with breakthroughs in autoimmune treatments, gene editing technologies, and targeted cancer therapies poised to alter therapeutic landscapes significantly.UCB has achieved another milestone with FDA approval for Kygevvi, an ultra-rare disease medication marking their third approval in rare conditions within three years. This success underscores UCB's strategic focus on niche markets that offer less competition but significant patient impact. Advancements in genetic research aSupport the show

"Society says it's better dead than disabled." This week, we sit down with Ryan Yellowlees , a counselor and activist who is living—and thriving—with Duchenne Muscular Dystrophy, decades past his initial prognosis. Ryan gets real about the progression of his condition , the systemic ableism he fights against , and why he's fiercely campaigning against the expansion of Medical Assistance in Dying (MAiD), calling it modern-day eugenics. He shares his unique perspective on finding strength and purpose in disability and why, given the choice, he'd live this life all over again.Find Ryan: https://www.lifeempoweredcounselling.caFollow Sickboy on Instagram, TikTok and Discord!

Episode 152: of the American Grown Podcast in the Colortech Creative Solutions studios with Lance Hains Clinical Specialist & Advocate for Duchenne Muscular Dystrophy.In this episode, we sit down with my cousin Lance Hains, a West Chester University graduate, Clinical Specialist, and passionate runner — for a powerful conversation about purpose, family, and perseverance.Lance opens up about his younger brother Levi, who lives with Duchenne Muscular Dystrophy (DMD), and how that diagnosis changed his outlook on life and fueled his drive to advocate for others.Recently completing the Chicago Marathon alongside his brother Luke, Lance reflects on how running has become both a personal mission and a metaphor for resilience — proving that life's toughest challenges can be met one mile at a time.Levi's Fight to END DUCHENNESHOW SPONSORS:College Knowledge Foundation. Your path to higher education.Cleona Coffee Roasters. A small batch coffee roastery & coffee shop, veteran & first responder owned located inside 911 Rapid Response in Annville PA.Angelo's Pizza. Enjoy mouthwatering Italian dinners.Triggered 22. Support a local veteran and help spread awareness for PTSD & #22aday.Modern Gent Customs. We don't make basics...We make statements.Hains Auto Detailing. Have your car smiling from wheel to wheel.A&M Pizza. Authentic Italian quality meals.Boyer's Tavern. Proper food & drinks made by slightly improper people.Hossler Engraving. Looking for unique handcrafted gifts for all occasions Zach has you covered.Sip or Snack break.SIP: Garage Beer.SNACK: Jurgy.OFFICIAL STUDIO SPONSOR: Colortech Creative Solutions takes your creative projects from visualization to realization. We've been doing so since 1980 all while keeping your budget in mind.To see photos of today's guest follow on social media:IG: AmericanGrownPodcastFB: American Grown Podcast or visits us at American Grown Podcast

"Society says it's better dead than disabled." This week, we sit down with Ryan Yellowlees , a counselor and activist who is living—and thriving—with Duchenne Muscular Dystrophy, decades past his initial prognosis. Ryan gets real about the progression of his condition , the systemic ableism he fights against , and why he's fiercely campaigning against the expansion of Medical Assistance in Dying (MAiD), calling it modern-day eugenics. He shares his unique perspective on finding strength and purpose in disability and why, given the choice, he'd live this life all over again.Find Ryan: https://www.lifeempoweredcounselling.caFollow Sickboy on Instagram, TikTok and Discord!

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Let's dive into the latest news shaping this dynamic industry.Bristol Myers Squibb recently made headlines with their acquisition of Orbital Therapeutics for a remarkable $1.5 billion. This strategic move is aimed at enhancing their in vivo cell therapy capabilities, particularly in treating autoimmune disorders. In vivo cell therapy is a pioneering approach that allows genetic modifications directly within a patient's body, potentially revolutionizing the treatment landscape for numerous conditions. This acquisition underscores Bristol Myers Squibb's commitment to pushing the boundaries of innovative cell therapy technologies and reflects a broader trend in the industry towards personalized medicine.In another significant development, AstraZeneca has aligned with the Trump administration's Most Favored Nation pricing program, agreeing to provide Medicaid drugs at prices competitive on a global scale. This decision marks a strategic shift towards cost reduction, especially in chronic disease management and respiratory therapeutics. The move is indicative of AstraZeneca's efforts to adapt to regulatory pressures and evolving policies that emphasize value-based healthcare delivery.Meanwhile, Ypsomed has announced plans to invest $248 million in establishing a manufacturing facility in North Carolina. This facility will focus on producing auto-injectors, essential for treating diabetes and metabolic disorders. The investment signifies a strategic operational expansion aimed at meeting rising demand in North America, highlighting the growing importance of drug delivery devices in the therapeutic landscape.Turning to clinical trials, Regeneron has unveiled promising Phase 1/2 data for its DB-OTO gene therapy targeting genetic hearing loss in children. By using AAV vectors to address DFNB9-related synaptic transmission deficits, this therapy could mark a breakthrough for those suffering from hereditary hearing conditions. Satellos has also presented encouraging Phase 1 results for SAT-3247, an oral small molecule targeting AAK1 in Duchenne muscular dystrophy, with plans to proceed to Phase 2 trials focused on muscle regeneration.In oncology, Taiho and Cullinan's Phase 2 data on zipalertinib showed efficacy against EGFR-mutated non-small cell lung cancer with brain metastases. This advancement highlights the potential of tyrosine kinase inhibitors in precision oncology. Similarly, Arcus Biosciences reported a median survival of 26.7 months for its combination therapy with domvanalimab and zimberelimab in gastroesophageal adenocarcinoma trials, underscoring the promise of TIGIT-targeted therapies.Assembly Biosciences has shared promising Phase 1b results for its ABI-5366 helicase-primase inhibitor, achieving an impressive 94% reduction in herpes simplex virus shedding. OS Therapies reported significant survival improvement with its OST-HER2 vaccine in recurrent pulmonary metastatic osteosarcoma patients, positioning HER2-targeting immunotherapies as promising cancer treatment interventions.Cabaletta Bio has made strides with its resecabtagene autoleucel CAR-T therapy, demonstrating B cell elimination without preconditioning in pemphigus vulgaris trials. This innovation opens new doors for autoimmune disease management through advanced cell therapies.On the business development front, Roche's out-licensing of its GLP-1/GIP agonist CT-388 to Chugai for diabetes and obesity treatment exemplifies strategic partnerships focused on addressing metabolic disorders through novel small molecules.The sector is also witnessing significant financial activities with Evommune filing an IPO to advance treatments for inflammatory conditions. Meanwhile, Quoin Pharmaceuticals raised $104.5 million through private placement to concentrate on rare disSupport the show

Dr. Neil Toupin, a child neurologist at UK HealthCare, shares valuable information about Duchenne muscular dystrophy for referring providers.

Shoot us a Text.Today, Paul and Chris bring you a story about Jacob Miller, a high school senior living with Duchenne Muscular Dystrophy. Jacob has big plans for being only eight years old and he just got a life-changing boost from our friends at West Herr Automotive: a wheelchair-accessible van that's opening the road to his future in engineering.Read the story here. Join Paul J Daly and Kyle Mountsier every morning for the Automotive State of the Union podcast as they connect the dots across car dealerships, retail trends, emerging tech like AI, and cultural shifts—bringing clarity, speed, and people-first insight to automotive leaders navigating a rapidly changing industry.Get the Daily Push Back email at https://www.asotu.com/ JOIN the conversation on LinkedIn at: https://www.linkedin.com/company/asotu/

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The FDA has rejected Capricor's therapy for Duchenne Muscular Dystrophy (DMD), causing shares to plunge due to insufficient evidence of effectiveness for cardiomyopathy associated with DMD. Lilly and Sarepta also received Complete Response Letters (CRLs) for their therapies. Moderna has won full approval for its COVID-19 vaccine for higher-risk kids, while Pacira has cut staff in San Diego. The FDA has released a cache of CRLs, providing insights into the journeys to market for various drugs. AbbVie has acquired Ichnos Glenmark's lead myeloma antibody, and Ultragenyx's stock plummeted after a late-stage trial disappointment. Kennedy postponed a preventive care panel meeting after a Supreme Court win. Transitioning from one piece of news to the next, let's move on to the webinar exploring digitization strategies for drug development. Stay tuned for more updates in the pharmaceutical industry.

Synopsis: When an introverted engineer becomes the President & CEO of REGENXBIO, transformation follows. Curran Simpson joins host Rahul Chaturvedi to unpack his unlikely journey from biotech operations to the C-suite—and how that hands-on experience is reshaping gene therapy's future. They dive into the evolution of REGENXBIO's pipeline, tackling ultra-rare diseases like MPS II, ambitious plans for Duchenne Muscular Dystrophy, and commercial partnerships with giants like AbbVie. Curran offers hard-earned leadership lessons, honest reflections on scaling science, and insights into how one-time gene therapies could revolutionize treatment in both rare and common diseases. From clinical nuance to strategic boldness, this is a masterclass in biotech leadership, platform focus, and staying patient-first—no matter how complex the science or market. Biography: Curran M. Simpson is the President and Chief Executive Officer and member of the Board of Directors at REGENXBIO. Mr. Simpson previously served as the Company's Chief Operating Officer. In that role, he led key business functions including Research & Clinical Development, Corporate Strategy, Manufacturing & Quality, Regulatory, and Commercial Operations. Mr. Simpson joined REGENXBIO in 2015 with extensive leadership experience across biopharmaceutical operations and served as the Company's Chief Technology and Operations Officer before becoming COO. Prior to joining REGENXBIO, he was the Regional Supply Chain Head for North America and Interim Chief Operating Officer at GlaxoSmithKline (GSK). Mr. Simpson earlier served as interim CEO of Human Genome Sciences (HGS), where he led the integration of HGS into GSK, and as Senior Vice President of Operations and Vice President of Manufacturing Operations at HGS. Prior to HGS, Mr. Simpson was Director of Manufacturing Sciences at Biogen. Earlier in his career, Mr. Simpson served in an overseas assignment at Novo-Nordisk Biochem in Denmark and in various senior development and engineer roles at Genentech, working on Herceptin and Avastin, among other roles. Mr. Simpson has an M.S. in surface and colloid science from Clarkson University and a B.S. in chemistry from the Clarkson College of Technology.

Welcome to Season 2 of the Orthobullets Podcast.In this episode, we review the high-yield topic of ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Duchenne Muscular Dystrophy⁠⁠⁠⁠⁠ from the Pediatrics section.Follow ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Orthobullets⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ on Social Media:⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Facebook⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Instagram⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Twitter⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠LinkedIn⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠YouTube

Episode 135: of the American Grown Podcast in the Colortech Creative Solutions studios with Luke Hains Spine Specialist & Advocate for Duchenne Muscular Dystrophy.In this episode, I sit down with my cousin Luke Hains, a spine specialist for VB Spine. Luke opens up about growing up in Lebanon, PA, and how his younger brother's battle with Duchenne Muscular Dystrophy shaped his purpose. From working in medical tech to running marathons and raising awareness, Luke's story is driven by one thing: showing up for the people you love.To learn more about Duchenne Muscular Dystrophy click here: https://www.mda.org/disease/duchenne-muscular-dystrophySHOW SPONSORS:College Knowledge Foundation. Your path to higher education.A&M Pizza. Authentic Italian quality meals.Boyer's Tavern. Proper food & drinks made by slightly improper people.Triggered 22. Support a local veteran and help spread awareness for PTSD & #22aday.Cleona Coffee Roasters. A small batch coffee roastery & coffee shop, veteran & first responder owned located inside 911 Rapid Response in Annville PA.Modern Gent Customs. We don't make basics...We make statements.Hains Auto Detailing. Have your car smiling from wheel to wheel.Hossler Engraving. Looking for unique handcrafted gifts for all occasions Zach has you covered.Angelo's Pizza. Enjoy mouthwatering Italian dinners.Take a sip or snack break.SIP: Garage Beer.SNACK: Jurgy.OFFICIAL STUDIO SPONSOR: Colortech Creative Solutions. Colortech Creative Solutions takes your creative projects from visualization to realization. We've been doing so since 1980 all while keeping your budget in mind.To see photos of today's guest follow our Social media: IG- https://www.instagram.com/americangrownpodcast/ FB-https://www.facebook.com/profile.php?id=100077655465940 or visits us at https://rss.com/podcasts/americangrownpodcast/

A protest is taking place at the Dáil this lunchtime, calling on the Health Minister to speed up the approval for and funding of a new drug for children with Duchenne Muscular Dystrophy. Families such as the Brogans have described the drug as a "game changer". To tell us more, we're joined by Conor Hunt...

Duchenne Muscular Dystrophy, or DMD as it is commonly known, is a progressive neuromuscular disease that causes the weakening of muscles over time.It affects almost exclusively boys, with symptoms typically appearing between the ages of one and three. In Ireland today, around 110 people are known to be living with DMD. Most of them are under the age of 18.Fianna Fáil Senator Teresa Costello has called on the European Commission, HSE and Minister of Health to regulate the drug Givinostat here for children with DMD.Joining Kieran to discuss is Senator Teresa Costello, as well as Úna and Eilish, both parents of children who suffer from Muscular Dystrophy.

About Andrea Andrea is a follower of Jesus, wife of Shawn, and mom of two boys; Sam (who has Duchenne Muscular Dystrophy) and Zach. As a younger adult, she obtained her Bachelor of Science in Civil Engineering, but left the workforce to homeschool her kids 15 years ago. Her family plays a small part in the special needs ministry 'Joni and Friends' via volunteering for their Wheels for the World program and enjoying their annual family camps. She's blessed to be mentor and friend to newer homeschool moms and to younger moms who are navigating the joys and the hard messiness of this special needs life. About the Episode In this conversation, Andrea Duerkop shares her journey as a special needs parent, discussing the challenges and triumphs of raising her son Sam, who has Duchenne muscular dystrophy. She reflects on the emotional journey of receiving the diagnosis, the importance of community support, and the lessons learned through faith and resilience. Andrea emphasizes the significance of sibling dynamics, the feeling of being loved and chosen, and the impact of giving back through service. The conversation highlights the importance of finding joy amidst challenges and the power of community in navigating the complexities of special needs parenting. Related Links By the Brook            

On this special episode of Talking Buffalo, Patrick Moran is joined by 16-year old Buffalo Bills superfan Tommy Parzymieso to discuss both his challenges and recent incredible moment, announcing the Bills first-round pick at the NFL Draft last week in Green Bay. The episode is recorded live from Imperial Pizza in South Buffalo.Born and raised in Orchard Park, Tommy at age 2 was diagnosed with Duchenne Muscular Dystrophy, a very rare genetic muscle wasting disorder affecting his muscles and heart as well as his lungs. As a result of his skeletal muscles being weak, he's reliant on an electric wheelchair full time to get around. But that hasn't stopped this young man's unbelievable spirt and love for sports.Through the Make-A-Wish Foundation, Tommy was along with his family was given a personalized tour of the Bills facility a couple of weeks before the draft. Tommy knew about that and that he'd be getting the chance to meet Sean McDermott and Brandon Beane. What he didn't know was that Beane was going to surprise him and ask Tommy to travel to Green Bay and announce Buffalo's first pick in front a huge live audience. Tommy documents the entire experience.Tommy also talks about his favorite team, why last year's first pick Keon Coleman is his favorite player, his dream of being a football coach someday, his family and plenty more.Tommy and his family will be hosting a "Team Tommy" fundraising event that takes place this Saturday, May 3rd, being held at the Wings Meeting Place in Orchard Park, not far from Highmark Stadium. Proceeds from the fundraiser will help improve modifications needed inside their home, specialized equipment and other resources that can help Tommy continue his quality of life for years to come.  Please consider supporting the event.Big shout-out and thank you once again to Imperial Pizza for being a gracious host and for their excellent wings. They now have two locations, 1035 Abbott Road and also 1665 Main Street♦♦♦♦♦Follow Patrick Moran/Talking Buffalo Podcast Substack: Patrick Moran's SubstackTwitter: @PatrickMoranTBBlue Sky: @PatrickMoranTB.bsky.socialInstagram: @PatrickMoranTBFacebook: Talking Buffalo Podcast 

What if the treatment they said was impossible… actually worked? In this inspiring episode of The Real Health Podcast, Dr. Ron Hunninghake, MD sits down with Blake Benton to discuss his family's incredible journey with stem cell therapy for Duchenne muscular dystrophy. Learn how Coming Together for a Cure, a nonprofit founded by the Benton family, is now helping hundreds of families access life-changing treatments!Learn more about our guest, Blake Benton:•Coming Together for a Cancer Cure: https://www.ctfac.org/•Watch The Sunshine Dreamer Documentary: https://www.ctfac.org/the-sunshine-dreamer-documentaryLearn more about the hosts:Dr. Ron Hunninghake, MD: https://riordanclinic.org/staff/ron-hunninghake-md/Interested in becoming a Patient:https://riordanclinic.org/request-an-appointment/Read the transcript:https://realhealthpodcast.orgLearn more about Riordan Clinic:https://riordanclinic.org/Thanks to This Episode's SponsorRiordan Clinic Nutrient Store: https://store.riordanclinic.org/Disclaimer: The information contained on the Real Health Podcast and the resources mentioned are for educational purposes only. They're not intended as and shall not be understood or construed as medical or health advice. The information contained on this podcast is not a substitute for medical or health advice from a professional who is aware of the facts and circumstances of your individual situation. Information provided by hosts and guests on the Real Health Podcast or the use of any products or services mentioned does not create a practitioner-patient relationship between you and any persons affiliated with this podcast.

Send us a textThis is a newly re-edited version of one of our first episodes from almost three years ago. It has been one of our most popular episodes, and we've given it a fresh new sound.  Co-hosts Dr. Janet Price and Gregg Kaloust sat down with dear friend Chris Balch to talk about our trek to Mt. Everest Base Camp with Everest to End Duchenne to raise awareness and funds for research to find a cure for Duchenne Muscular Dystrophy. We are stll hoping for a cure. We hope you enjoy our conversation.Gregg Kaloust is writing a memoir of that trek and others he made with Chris, Everest to End Duchenne, and other friends. He is posting excerpts along with other writings on his substack at https://greggkaloust.substack.com/publish/homeYou can learn more about Duchenne and efforts to find a cure at https://everesttoendduchenne.org. There's a link there to the documentary of that first trek.Support the showConnect with Janet at https://drjanetprice.comGregg has a new substack newsletter where he's publishing writings old and new: poems, short pieces, works in progress, opinions and notes.You can email Gregg at gregg@kannoncom.com Gregg wears Tyrol pickleball shoes, the only company that makes shoes just for pickleball. He has been wearing the same pair of Velocity V model shoes for almost a year, and he plays a lot! Click here to purchase Tyrol Pickleball shoes (note, if you purchase Tyrol pickleball shoes after clicking this link Oldish may receive a commission. Thanks for helping to support our podcast!)Comments, suggestion, requests: oldish@kannoncom.comThanks to Mye Kaloustian for the music.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Sage has rejected Biogen's unsolicited buyout offer and will seek strategic alternatives. Biogen and Eisai have received approval for a monthly maintenance regimen for Alzheimer's drug Leqembi. Veru's drug has shown promise in sparing lean mass in overweight adults on Wegovy. The Duchenne muscular dystrophy space is on the verge of a pivotal era with several companies developing investigational therapies. Akero has rebounded in a mid-stage trial, Daiichi Sankyo is optimistic for 2025, and Allakos is cutting its workforce. The text discusses the advancements in the Duchenne Muscular Dystrophy (DMD) space as it enters a pivotal era, with companies such as Capricor Therapeutics, Wave Life Sciences, and RegenxBio working on investigational therapies to address unmet needs. It also mentions Daiichi Sankyo's recent success with the approval of Astrazeneca-partnered Dato-DXD. Additionally, new treatments are in development for Achondroplasia to challenge Biomarin's Voxzogo. Novo's obesity drug shows promising results, Merck's Keytruda combo fails in a Phase III trial for GI cancer, and Tris Pharma wins late-stage for a non-opioid painkiller. The text also invites feedback from readers on topics they would like to see covered in the future.

Join Elijah Stacy as he explores the groundbreaking work of Somite Therapeutics, where AI and cell therapy collide to redefine medicine. In this special episode, hear from industry trailblazers like Dr. Micha Breakstone (Chorus.ai, NeuraLight.ai), Dr. Jonny Rosenfeld (MIT FutureTech), and Dr. Kristy Brown (Solid Biosciences, Rejuvenate Bio) as they discuss Somite's AI-powered platform, AlphaStem, and its potential to transform drug development and treat complex diseases like Duchenne Muscular Dystrophy. Discover how cutting-edge science, innovation, and a bold vision for the future of healthcare are paving the way for revolutionary breakthroughs.

  It's hard to fathom why certain children receive life altering diagnoses. Pediatricians simply want to help, and that help usually comes in the form of early diagnosis. Duchenne muscular dystrophy, otherwise known as Duchenne or DMD, is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Early screening and identification can mean quicker access to care, early intervention programs and special education resources. Thanks to early diagnosis and advances in care, life expectancy is increasing.  Our guest joining us to discuss diagnosing and treating DMD is Michele Yang, MD, who specializes in pediatric neurology and neuromuscular medicine at Children's Hospital Colorado. She is also an associate professor of pediatric neurology at the University of Colorado School of Medicine.  Some highlights from this episode include:  How diagnosis of DMD has changed over the years.  Understanding the new, advanced treatments that exist.  Tips and tricks for primary care providers to utilize.  Realistic outcomes for these kids with DMD.  For more information on Children's Colorado, visit: childrenscolorado.org. 

Around 30% of boys diagnosed with Duchenne muscular dystrophy also experience cognitive dysfunction and neurodevelopmental disorders like autism and ADHD. A UT Health San Antonio neuroscientist is doing research he hopes will uncover what is causing these deficits and how they might be treated.

Initially aspiring to become a doctor like his parents and later venturing into the business world, Mazi Keyghobadi experienced significant failures and personal confusion. This led to an awakening five years ago, where he realized that external achievements did not bring him true happiness. Instead, he found his purpose within nonprofit work, driven by his son's Duchenne Muscular Dystrophy diagnosis. Welcome back to another enlightening episode of the Better Call Daddy Show! I'm your host, Reena Friedman Watts. Today, we dive deep into the transformative world of magic mushrooms, meditation, and muscular dystrophy with our special guest, Mazi Keyghobadi. As always, our insightful co-host, Wayne "Big Daddy" Friedman joins us. In this episode, Mazi shares his profound experiences with magic mushrooms, describing them as a "laxative for the soul," capable of sweeping away inner negativity and trauma. We'll hear how these natural substances have not only improved his mental clarity but also enhanced his roles as a father and nonprofit founder of Hope for Luka. Mazi likens the impact of psychedelics to the intriguing effects shown in the movie "Limitless" and discusses the significance of preparation and meditation when using these powerful tools. We'll also explore Mazi's mission to introduce the benefits of magic mushrooms to the muscular dystrophy community, his journey from aspiring doctor to impactful nonprofit leader, and how a life-altering "awakening" helped him find true happiness and purpose. Plus, Mazi will share inspiring anecdotes about thought leaders like Paul Stamets and Eckhart Tolle, as well as his commitment to authenticity and societal betterment. Stay tuned as we unravel Mazi Keygohbadi's story of self-discovery, emotional healing, and unwavering dedication to meaningful change. This is an episode you won't want to miss! Unfortunately, I don't have the exact timestamps for this episode. Instead, I'll provide a suggested timeline breakdown for "Better Call Daddy: Mazi Final Audio" based on the key facts discussed. You can adjust as needed when you have the precise timing from the episode.   (00:00 - 01:00) - Introduction - Introduction of the host Reena Watts, the guest Wayne Friedman (Big Daddy), and the guest Mazi Keyghobadi. - Brief overview of what to expect from the episode. (01:00 - 05:00) - Mazi's Background - Mazi's emigration from Iran, early life, and initial aspirations to become a doctor. - His decision to pivot away from a medical career towards entrepreneurship and eventual nonprofit work. (05:00 - 10:00) - Personal Awakening - Mazi discussing his midlife crisis and awakening circa five years ago. - The impact of Eckhart Tolle's "The Power of Now" on his transformation. (10:00 - 15:00) - Magic Mushrooms and Self-Healing - Mazi's first experience with magic mushrooms. - The comparison to the movie "Limitless" and the preference for natural substances over synthetic ones like LSD. (15:00 - 20:00) - Healing Through Psychedelics - Study involving an OCD patient in a sensory deprivation "dark room." - Mazi's description of mushrooms as a "laxative for the soul." (20:00 - 25:00) - Application of Personal Healing - Using mushrooms to overcome negativity and trauma. - Mazi's improvement in his role as a father and an advocate through his nonprofit, "Hope for Luca." (25:00 - 30:00) - Societal and Personal Impacts - Challenges of managing a nonprofit with limited resources. - Persistence and remaining true to values despite financial and political challenges. (30:00 - 35:00) - Psychedelics and the Brain - How psilocybin from magic mushrooms interacts with the brain. - Potential therapeutic applications, including microdosing for stress management. (35:00 - 40:00) - Practical Advice and Reflections - Importance of meditation and preparation before using psychedelics. - Reflections on personal and societal transitions, stress, and finding fulfillment beyond material wealth. (40:00 - 45:00) - Nonprofit Fundraising and Advocacy - Details on the new fundraising campaign for research on exons 5 to 26. - The significance of Mazi's son's condition as a motivator for his work. (45:00 - 50:00) - Final Thoughts - Wayne Friedman's reflections on spirituality and genuine compassion in the medical field. - Reena Watts wrapping up the segment, encouraging audience engagement. Connect with Mazi and support his mission at hopeforluka.org, and be part of the movement that champions the extraordinary lives of children like Luka. We'd love to hear from you. Drop us a review—reviews help more people find the show—and let us know what you like and what you'd like us to explore next. Share this episode with someone who could be inspired by Mazi's story and help spread hope for Luka and many others facing similar challenges. If you liked this story check out my first episode with Mazi here https://youtu.be/4FMsFkJ3UAQ?si=wqunjY8NJscrdkZA Connect with Reena instagram.com/reenafriedmanwatts linkedin.com/in/reenafriedmanwatts twitter.com/reenareena https://www.youtube.com/@BetterCallDaddy https://www.facebook.com/groups/BusinessLaughsandLinkedin   Special Shoutout to the Daddies Podcast for editing this episode!  

Negotiation is a complex art and science. It's not about negotiating better; it's about becoming a better negotiator.  Enter Lousin Mehrabi, one of the world's top professional negotiators, with expertise spanning billion-dollar IPOs, mergers, and even peace-building efforts. As a seasoned advisor and trainer, Lousin has made it her mission to equip individuals and organisations with the tools to navigate complex negotiations and achieve transformative results. Today, she's an internationally sought-after keynote speaker and a global head of negotiation training for ADN Group. She's also a special advisor to C-Suite executives for negotiation strategy and personal growth. Now, with all her qualifications and knowledge, she may come across as a tough business negotiator. And the truth of it is that she is. She understands this world inside and out - but she is also very vulnerable.  Beyond her professional expertise, Lousin opens up about personal challenges, including negotiating through the hardest moments of her life - her son's diagnosis with Duchenne Muscular Dystrophy, an incurable disease. Her journey is a testament to resilience, emotional intelligence, and the power of having peace negotiations with yourself. 03:36 - What does a professional negotiator do? 12:04 - Negotiation as a male-dominated industry 24:30 - Training negotiators 29:19 - Negotiations in conflict zones 32:31 - Tactics in negotiations 37:07 - Spotting manipulation in negotiations 39:04 - Walking away from a deadlock 41:35 - Lousin's negotiation experience 46:27 - Negotiating with yourself 51:31 - The path to world peace Show Sponsors: Allsopp & Allsopp: Redefining real estate, through cutting-edge technology and setting new standards for seamless, elevated customer experience. Keep moving with Allsopp & Allsopp.https://bit.ly/Allsopp-and-Allsopp Socials: Spencer Lodgehttps://www.instagram.com/spencer.lodge/?hl=en https://www.tiktok.com/@spencer.lodge https://www.linkedin.com/in/spencerlodge/ https://www.youtube.com/c/SpencerLodgeTV https://www.facebook.com/spencerlodgeofficial/  Lousin Mehrabihttps://www.linkedin.com/in/lousin-mehrabi/?originalSubdomain=ae https://www.instagram.com/lousinmehrabi/https://www.youtube.com/@lousinmehrabi

Episode 106: of the American Grown Podcast in the Colortech Creative Solutions studios with Jon Simpson Personal care Assistant at Cedar Crest High School.-In this episode Jon shares with us his journey to become a personal care assistant. He currently works at Cedar Crest high school where he helps my cousin Levi Hains who was born with Duchenne Muscular Dystrophy a rare genetic disease that affects the muscles. Jon and I also talk about the culture at Cedar Crest and his passion for Penn State sports. Lastly with the holiday season just around the corner we discuss how being present & nice can make a positive impact in the world.-To learn more about Levi's Fight to END Duchenne click here: https://www.facebook.com/LevisFight-#ad this episode is brought to you by these sponsors:Triggered 22. Support a local veteran and help spread awareness for PTSD & #22aday.Cleona Coffee Roasters. A small batch coffee roastery & coffee shop, veteran & first responder owned located inside 911 Rapid Response in Annville PA.Colortech Creative Solutions. Colortech Creative Solutions takes your creative projects from visualization to realization since 1980 while keeping your budget in mind.-To see photos of today's guest follow our Social media: IG- https://www.instagram.com/americangrownpodcast/ FB-https://www.facebook.com/profile.php?id=100077655465940 or visits us at https://rss.com/podcasts/americangrownpodcast/

Following the announcement of Labour's first budget in fourteen years – and the first Budget announced by a woman Chancellor – we hear how women across the country will be affected. Anita Rani speaks to Mehreen Khan, the economics editor of The Times, Sara Reis, deputy director and head of research at Women's Budget Group and Jo, a participant in the Changing Realities project, a collaboration of parents and carers on a low income and researchers from the University of York and Child Poverty Action.With Halloween upon us, Queen of African Horror Nuzo Onoh joins Anita to talk about her new novel Where the Dead Brides Gather, her journey to getting published and spooky celebrations in Nigeria. How many of us have grown up with parts of our lives unknown to our parents? Born with a degenerative muscular disease, Duchenne Muscular Dystrophy, Mats Steen was in a wheelchair by the age of 8, and was in an out of hospital right up until he died at the age of 25. As his family mourned his loss, it emerged that Mats had an online life that his parents knew nothing about. In a new Netflix documentary, his mother Trude, his father Robert and his sister Mia describe what it was like to discover Mats' secret world. Trude joins Anita to discuss. Presenter: Anita Rani Producer: Laura Northedge

Our guest this week is Nate Plasman of Lombard, IL, a vice president at family owned Monroe Transportation and father of three, including a son with a rare genetic disease.Nate and his wife, Sara, have been married for 20 years and are the proud parents of three children: Grace (15), Jackson (12) and Andrew (10) who has Duchenne Muscular Dystrophy, a progressive and severe muscle-wasting condition that typically results in premature death.Duchenne impacts around 15,000 individuals in the United States, predominantly males, and over 300,000 globally. It is a condition that transcends cultural, economic, and social lines, affecting families worldwide.We learn about a host of organizations seeking cures and improving the lives of those living with muscular dystrophy, including; Parent Project MD, Cure Duchenne and Serepta Therapeutics. It'a an uplifting story about faith, family and overcoming adversity all on this episode of the SFN Dad to Dad Podcast. Show NotesPhone – (630) 248-3220Email - nate@monroe-trans.comLinkedIn - https://www.linkedin.com/in/nathan-plasman-0a050149/Facebook - https://www.facebook.com/nathan.plasman/friendsMonroe Transportation – https://monroe-trans.com/Parent Project MD - https://www.parentprojectmd.org/Cure Duchenne - https://cureduchenne.org/Serepta Therapeutics - https://www.sarepta.com/Special Fathers Network - SFN is a dad to dad mentoring program for fathers raising children with special needs. Many of the 800+ SFN Mentor Fathers, who are raising kids with special needs, have said: "I wish there was something like this when we first received our child's diagnosis. I felt so isolated.  There was no one within my family, at work, at church or within my friend group who understood or could relate to what I was going through."SFN Mentor Fathers share their experiences with younger dads closer to the beginning of their journey raising a child with the same or similar special needs. The SFN Mentor Fathers do NOT offer legal or medical advice, that is what lawyers and doctors do. They simply share their experiences and how they have made the most of challenging situations.Check out the 21CD YouTube Channel with dozens of videos on topics relevant to dads raising children with special needs - https://www.youtube.com/channel/UCzDFCvQimWNEb158ll6Q4cA/videosPlease support the SFN. Click here to donate: https://21stcenturydads.org/donate/Special Fathers Network: https://21stcenturydads.org/  

Join us for a fantastic conversation with an expert in the field of neuromuscular disorders, Dr. Kevin Flanigan. Dr. Flanigan is the director of the Center for Gene Therapy at Nationwide Children's Hospitals, which developed the first FDA-approved gene therapy to treat Duchenne Muscular Dystrophy. He teaches us when to include muscular dystrophy in the differential, how BiPAP extends the lives of patients with Duchenne, and what the latest gene replacement therapies have to offer. 

Elijah Stacy, founder of Destroy Duchenne, sits down with Frank Gleeson, co-founder and CEO of Satellos Bioscience, to explore the vision and leadership that have driven Satellos to the forefront of regenerative medicine. In this episode, we dive into Frank's personal journey, the foundational ideas behind Satellos, and the pivotal moments that shaped the company's mission to revolutionize treatments for muscular dystrophy. This conversation highlights the strategic decisions and innovations that continue to propel Satellos toward its goal of pioneering novel therapies for Duchenne Muscular Dystrophy. Learn More about Satellos: https://satellos.com Want to be on the podcast? Click here: https://www.destroyduchenne.org/podcast Learn more about the Destroy Duchenne Podcast here: https://www.destroyduchenne.org/podcast Website: https://www.destroyduchenne.org Social Media: Facebook: https://www.facebook.com/destroyduchenne Instagram: https://www.instagram.com/destroyduchenne Twitter: https://twitter.com/destroydisease LinkedIn: https://www.linkedin.com/company/destroyduchenne ABOUT DESTROY DUCHENNE PODCAST: The Destroy Duchenne Podcast is a platform dedicated to telling the stories of those affected by Duchenne Muscular Dystrophy (DMD), a devastating muscle-wasting disease. Hosted by Elijah Stacy, a 23-year-old founder of Destroy Duchenne and a DMD patient himself, this podcast seeks to bring attention to the fight against DMD by interviewing those closest to the disease—patients, families, doctors, and researchers. Destroy Duchenne is a registered 501(c)(3) nonprofit organization with a mission to complete the cure for Duchenne Muscular Dystrophy by advancing medical technologies into human practice. Federal Tax ID: #82-3268952

Our guest this week is Mazi Keyghobadi of Houston, TX, a father and outspoken advocate for those with disabilities. Mazi, who was born and raised in Iran, emigrated to the U.S. as a teenager with his parents and brothers in 2000. Mazi and his x-wife, Dunia, were married for nine years, before divorcing in 2021 and are the proud parents of son, Luka (7) who has Duchenne Muscular Dystrophy.Mazi has also formed Hope For Luka, a non-profit dedicated to funding Muscular Dystrophy research with the objective of finding a cure as well as advanced therapies for those impacted by Muscular Dystrophy. It's a an uplifting story about overcoming adversity, a father's commitment to his son, and advocacy for others.  All on this episode of the SFN Dad to Dad Podcast. Special Fathers Network - SFN is a dad to dad mentoring program for fathers raising children with special needs. Many of the 800+ SFN Mentor Fathers, who are raising kids with special needs, have said: "I wish there was something like this when we first received our child's diagnosis. I felt so isolated.  There was no one within my family, at work, at church or within my friend group who understood or could relate to what I was going through."SFN Mentor Fathers share their experiences with younger dads closer to the beginning of their journey raising a child with the same or similar special needs. The SFN Mentor Fathers do NOT offer legal or medical advice, that is what lawyers and doctors do. They simply share their experiences and how they have made the most of challenging situations.Check out the 21CD YouTube Channel with dozens of videos on topics relevant to dads raising children with special needs - https://www.youtube.com/channel/UCzDFCvQimWNEb158ll6Q4cA/videosPlease support the SFN. Click here to donate: https://21stcenturydads.org/donate/Special Fathers Network: https://21stcenturydads.org/ 

What's life?  What kind of world are we leaving for our children?  Mazi Keyghobadi shares his battle against muscular dystrophy for his son's future.  He believes you need to speak your mind and contribute!  He is leading by example. There's only one other person that has the rare form of muscular dystrophy that Mazi Keyghobadi's son has.  It wakes up part of you you never knew existed.  In this heart-stirring episode of Better Call Daddy, we meet Mazi Keyghobadi, a father whose love knows no bounds. Mazi is the co-founder of Hope for Luca, a nonprofit organization named after his seven-year-old son, who battles a rare form of muscular dystrophy. With the tenacity of a warrior and the heart of a lion, Mazi shares his family's journey through the trials and tribulations of seeking a chance for Luca to live a normal life.   Mazi's story is one of sacrifice, determination, and the relentless pursuit of hope. From selling his car to pouring his life savings into research, he embodies the spirit of a parent who will stop at nothing to give his child a fighting chance. His quest leads him to the forefront of scientific research, where he collaborates with leading experts and navigates the complex world of fundraising and advocacy.   But this episode is more than just a tale of a father's love. It's a call to action for anyone with a dream, a reminder that sometimes the most significant victories come from the heart. Mazi's message is clear: see the strength in those who fight battles unseen, hear their voices, and appreciate the heroes who live among us.   Join Reena Friedman Watts as she delves into the depths of a father's soul, exploring the power of unwavering faith and the beauty of a bond that defies all odds. Better Call Daddy: The Safe Space for Controversy.   Connect with Mazi and support his mission at hopeforluca.org, and be part of the movement that champions the extraordinary lives of children like Luca.   Go Fund Me page https://www.gofundme.com/f/raising-130k-to-fund-preclinical-research-study   Mazi on Linkedin https://www.linkedin.com/in/mazi-keyghobadi-55a92493/   PPMD (Parent Project Muscular Dystrophy) https://www.parentprojectmd.org TREAT-NMD is a global network of experts in the neuromuscular field https://www.treat-nmd.org/   Better Call Daddy: The Safe Space for Controversy.   Connect with Reena: https://bettercalldaddy.com https://linkedin.com/in/reenafriedmanwatts https://twitter.com/reenareena https://instagram.com/reenafriedmanwatts https://instagram.com/bettercalldaddypodcast   We'd love to hear from you. Drop us a review—reviews help more people find the show—and let us know what you like and what you'd like us to explore next.   Share this episode with someone who could be inspired by Mazi's story and help spread the hope for Luca and many others facing similar challenges.   Show notes created by https://headliner.app   ---  

In this episode, we review the high-yield topic of⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Duchenne Muscular Dystrophy⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠from the Pediatrics section. Follow ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Orthobullets⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ on Social Media: ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Facebook⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Instagram⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Twitter⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠LinkedIn⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠YouTube --- Send in a voice message: https://podcasters.spotify.com/pod/show/orthobullets/message