Podcasts about Duchenne 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 

Fighting a tough battle with Duchenne Muscular Dystrophy, Duncan needed help to breathe more comfortably. Thanks to Hot Cares, he's getting the life-changing medical support he needs—plus a little something extra to brighten his world. Hot Cares

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.

Guest: Paula Naughton

  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/

Mieko Hester Perez is an Advocate for families who have members with special needs. In 2009, she decided to go public with her son's success with medical cannabis and since then she has become a source of inspiration and information for families looking to explore alternative and holistic treatment options within the autism spectrum. The combination of 20 years of legal experience and over 17 years of special education knowledge, make Mieko a modern-day “warrior mom”. She has received special recognition from the United States Congress for her many hours of community service and dedication. Ms. Perez also received the Lifetime Advocate Award at the Chalice Festival for her efforts within the cannabis industry. Her son Joey was diagnosed with Duchenne Muscular Dystrophy in late 2011 in addition to his autism and passed away in April 2018 at the age of just 19 years old. Since that time Ms Perez has dedicated her life to serving families. This is just part of her story.

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

Another fortnight has passed, and it's another new episode. This week, we chat all things EIHL, including Dops decisions and video reviews, CHL action for the Sheffield Steelers, fitting tributes in the NHL, and, of course, the first of the Halloween-themed jerseys. Plus, special guest Amie McClean joins us to chat all things fundraising for her brother Ethan and Duchenne Muscular Dystrophy. Why not check out the video version of the podcast on our YouTube channel at www.youtube.com/@door14hockey Title music - Relapse by Fallen to Flux, Feb 04, 2015. Licensed under a Attribution-NoDerivatives 4.0 International License. The post Episode 123 – SHOOT!! appeared first on Door 14 Hockey.

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/  

In this episode, I am joined by Antoinette Stubbs to discuss her memoir Braveheart. The release of this podcast today celebrates Antoinette starting a new role n the British national Health Service today! In Braveheart, Annie narrated the experience of her son, Calum, who suffered with Duchenne Muscular Dystrophy. Our discussion highlights the early symptoms of the disease, the delay in diagnosis, and the burden of care. Just as the podcast highlights the grief that came with losing Calum, it also brings out the positivity that enables Annie to say that there is still much beauty in the world.

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. 

In Part 1, we heard about the bold and courageous approach that Ryan Benton's family took to treat his Duchenne Muscular Dystrophy. After seeing the success of the stem cell therapy, Blake and Ryan Benton felt very deeply that they needed to inform and educate others about this approach. From the grass roots of a local rock concert grew Coming Together for a Cure (CTFAC).The mission of CTFAC is to make stem cell therapy the standard of care for Duchenne Muscular Dystrophy a wide variety of rare and debilitating diseases. This is a challenging objective. Investigators need to be recruited, scientific evidence needs to be accumulated, and regulatory requirements need to be met. Because of the history of stem cell research in the US, all of these steps need to be done with utmost care and professionalism. Learn more at CTFAC.orgMentioned in this episode:Sunshine Dreamer AdWe are excited to share a late-breaking announcement from today's guest. Coming soon: The feature documentary, The Sunshine Dreamer, shares the inspiring story of Ryan Benton's journey battling Duchenne Muscular Dystrophy and becoming the first person with DMD to be treated with stem cell therapy, starting in 2008. Thanks to the therapy, his life expectancy nearly doubled, allowing him to continue his passion as a musician and inspiring thousands of others fighting to overcome their diseases. The film also showcases stories of families across the globe who Blake and Ryan helped to receive stem cell therapy through their nonprofit Coming Together for a Cure. Additionally, you will hear from celebrities and professional athletes who have become advocates for stem cell therapy based on their personal experiences receiving therapy. They believe this film will motivate people about the immense potential of stem cell therapy to become part of the standard of care for those battling rare diseases and beyond. You can watch the trailer at CTFAC.org. Coming Together For a CureInvitation to Check Out The AtlasThe Atlas

Paul Heaton and Nick Tyler on Calves to Cure Sale to benefit a cure for Duchenne Muscular Dystrophy, at BLS on September 19th.

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

Blake Benton's earliest memories were his brother declining due to Duchenne Muscular Dystrophy.About 10 years later, his parents and brother made a bold move. They went to Costa Rica to get an experimental stem cell therapy. And it worked. Ryan was able to live until he was 37 years old with little degeneration caused by his DMD. He died in 2023 of complications from kidney stones.Ryan and Blake started a non-profit named after a benefit concert they held – Coming Together for a Cure (CTFAC). The goal of CTFAC is to advocate for more stem cell research in DMD and other diseases. There are many challenges, especially in the US.We discussed:1. How his parents raised the boys and their sister2. The impact of Ryan's DMD on Blake's life and outlook3. The good fortune for Blake's parents to have grown up with a stem cell researcher who thought this could make a difference for Ryan.Mentioned in this episode:Sunshine Dreamer AdWe are excited to share a late-breaking announcement from today's guest. Coming soon: The feature documentary, The Sunshine Dreamer, shares the inspiring story of Ryan Benton's journey battling Duchenne Muscular Dystrophy and becoming the first person with DMD to be treated with stem cell therapy, starting in 2008. Thanks to the therapy, his life expectancy nearly doubled, allowing him to continue his passion as a musician and inspiring thousands of others fighting to overcome their diseases. The film also showcases stories of families across the globe who Blake and Ryan helped to receive stem cell therapy through their nonprofit Coming Together for a Cure. Additionally, you will hear from celebrities and professional athletes who have become advocates for stem cell therapy based on their personal experiences receiving therapy. They believe this film will motivate people about the immense potential of stem cell therapy to become part of the standard of care for those battling rare diseases and beyond. You can watch the trailer at CTFAC.org. Coming Together For a CureInvitation to Check Out The AtlasThe Atlas

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/ 

Making Connections and Changing LivesIn this episode of the True Tales by Disability Advocates Podcast, hosts MsBoye, Jennifer McKinney, and Adam Griebel present stories from disability advocates. The episode includes a reflection on Mesko's early advocacy and a reading of his story "Glen," followed by an interview with advocate Kyle Cox, highlighting the importance of community connections and perseverance. Guest host Toby Al-Trabulsi engages with Kyle about his advocacy journey, emphasizing the need for self-advocacy and the role of supportive relationships.Podcast logo ID:   Bottom left Mesko wearing glasses and a flat cap smiles into the camera. Bottom right Kyle Cox male with short brown hair smiling, seated wearing a light blue button-down shirt, red tie. All above TRUE TALES BY DISABILITY ADVOCATES SEASON 3 Art Spark TexasGuest Bio Kyle Cox graduated from Texas A&M in 2018 with a degree in Political Science, then received his Master of Public Administration from the Bush School of Public Service and Government in 2020.  Born in El Paso, Texas with Duchenne Muscular Dystrophy, a progressive muscle disorder that is the leading fatal genetic disorder in children, Kyle has learned to navigate a world not designed for people with disabilities.   He has fully immersed himself in both the disability and the “abled” communities and has adopted the mantra “It's not the disability, it's the ABILITY” choosing to focus on his abilities.While at Texas A&M, Kyle was involved in numerous campus organizations including the Diversity Commission, Beta Upsilon Chi (BYX) Christian fraternity, Aggie Adaptive Sports, the Bush School Ambassador's Council, the Symphonic Band, and the Hullabaloo Pep band.   He was twice elected to the Student Senate and was elected to the Bush School Ambassador Council.  He was also Chairman and Founder of the Disability Subcommission.Kyle served on the Student Advisory Board for Disability Services and the planning committees for the Student Services Building and the 21st Century Classroom Building.   Kyle served as a delegate for the MSC Fall Leadership Conference and the Abbott Family Leadership Conference and has worked with many Texas A&M officials, Disability Services, and the Center on Disability and Development to improve inclusion and accessibility on the A&M campus.  In the Spring of 2018, Kyle received the Gary Grey Accountability, Climate, and Equity (ACE) Award for his work in advocacy, and in the Spring of 2020, the Buck Weirus Award. Upon graduation, Kyle chose to remain in Aggieland and launched his own consulting company, Cox Consultants, which offers policy consulting and analytics to businesses and agencies.Links:https://www.facebook.com/KylesKomrades/https://kylecoxeportfolio.weebly.com/https://www.facebook.com/CoxConsultants/Videos:https://vimeo.com/269928141/236823631https://vimeo.com/269928141/269928141Highlights00:43 Welcome01:23 Episode Intro03:10 Mesko Intro06:32 Story - "Getting Older" by Mesko07:44 Hosts & Chris Chat07:47 Phenomena, not Conclusion12:15 Toby Welcomes Kyle14:02 Story - "Advocacy, Education, and Relationships" by Kyle Cox18:32 Toby & Kyle Chat18:32 Being a Self-Advocate22:23 Creating a Community & Being of SerClick here to listen with the full transcript.For more details about our programs go to Art Spark Texas, True Tales Podcast Page.

Join me in "The Power of Hope: Joshua Busby's Journey" for an inspiring conversation with Joshua Busby, also known as "The Cookie Monster" due to his raspy voice from using a non-invasive mechanical ventilation machine. Joshua shares his remarkable story of living with Duchenne Muscular Dystrophy and how he has transformed his life through faith, courage, and an unwavering sense of hope. Discover how he transitioned from being a former minister to a life coach, and the profound impact fatherhood has had on his journey. Learn about the mental and emotional resilience required to face his condition, and the power of community support in overcoming adversity. Joshua's story is a testament to the strength of the human spirit and the transformative power of hope. Joshua on X: https://x.com/joshuabusby Support Duchenne Muscular Dystrophy research: https://www.duchenneuk.org/ https://thoughtfullymindless.com/ Support the show by making a purchase from Fractal Zoo: https://fractalzoo.net/ Join the Thoughtfully Mindless newsletter here: https://t.co/PpVB5M7aex And the Thoughtfully Mindless community on X here:

As part of the 2024 Developmental Disabilities Conference, Dr. Peter Kang, Professor of Genetics at the University of Minnesota, presents a detailed summary of recent advances in gene therapy for children with neuromuscular disorders, specifically Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, and Pompe Disease. He uses case studies to demonstrate diagnostic dilemmas, intricacies of developing and delivering treatment, and the importance of shared decision making about therapies with families. Series: "Developmental Disabilities Update" [Health and Medicine] [Show ID: 39747]

As part of the 2024 Developmental Disabilities Conference, Dr. Peter Kang, Professor of Genetics at the University of Minnesota, presents a detailed summary of recent advances in gene therapy for children with neuromuscular disorders, specifically Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, and Pompe Disease. He uses case studies to demonstrate diagnostic dilemmas, intricacies of developing and delivering treatment, and the importance of shared decision making about therapies with families. Series: "Developmental Disabilities Update" [Health and Medicine] [Show ID: 39747]

As part of the 2024 Developmental Disabilities Conference, Dr. Peter Kang, Professor of Genetics at the University of Minnesota, presents a detailed summary of recent advances in gene therapy for children with neuromuscular disorders, specifically Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, and Pompe Disease. He uses case studies to demonstrate diagnostic dilemmas, intricacies of developing and delivering treatment, and the importance of shared decision making about therapies with families. Series: "Developmental Disabilities Update" [Health and Medicine] [Show ID: 39747]

Paul Heaton and his family are going to be holding their annual all class cattle sale at Billings Livestock on September 19th, with cattlemen having a chance to donate all or a portion of their proceeds from their cattle checks to the Calves to Cure DMD fund.  In addition, the Heaton family is hosting a raffle through the summer with participants having the chance to win one of eight, 50 pound boxes of beef.  Details can be found here: https://cureduchenne.org/calvestocure/ See omnystudio.com/listener for privacy information.

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

As if Orna Therapeutics' CEO Thomas Barnes, CEO isn't a enough to draw you in to the Business of Biotech, we teamed up with Advancing RNA Editorial & Community Director Anna Rose Welch to co-host this week's episode. Together, Anna Rose and I press Dr. Barnes on his transition from academia to biotech, the therapeutic proposition of circular, or "O" RNA and why it holds great potential to best linear RNA constructs, the novel, platform- and partnership-based approaches Orna is taking to address B-cell lymphomas and Duchenne Muscular Dystrophy, viral vector and lipid nanoparticle complexity, and a whole lot more.You've listened along for years -- now you can watch along, too! Go to bioprocessonline.com/solution/the-business-of-biotech-podcast, where you can put faces to voices as you watch hundreds of interviews with the world's best biotech builders. While you're there, subscribe to the #BusinessofBiotech newsletter at bioprocessonline.com/bob for more real, honest, transparent interactions with the leaders of emerging biotech. It's a once-per-month dose of insight and intel that you'll actually look forward to receiving! Check it out at bioprocessonline.com/bob!

This podcast episode dives into the screening and management of Duchenne muscular dystrophy, as well as diversity in genetic databases, and treating neuromuscular disorders. Jonathan is joined by Madhuri Hegde, Senior Vice-President & Chief Scientific Officer at Revvity, Inc., to talk about innovation in genomic testing. Use the following timestamps to navigate our episode!   (00:00)-Introduction  (03:54)-Entering into genomic technologies (05:13)-Duchenne muscular dystrophy (10:47)-Revvity and genome sequencing (13:13)-Ultrarapid genomic testing (urWGS) (15:18)-LANTERN project (17:42)-Lack of diversity in genetic databases (20:06)-New developments in genetics (22:19)-COVID-19 testing laboratories (27:27)-Madhuri's wishes for healthcare

Elijah Stacy, founder of Destroy Duchenne, and Dr. Michael Rudnicki, co-founder of Satellos Bioscience and Royal Society inductee, discuss Satellos' novel approach to muscle regeneration, focusing on stimulating the body's internal stem cells rather than using external transplants. This pioneering work has the potential to transform the future of treatment for Duchenne Muscular Dystrophy. Learn More about Satellos:https://satellos.com  Want to be on the podcast? Click here: https://www.destroyduchenne.org/podca... Learn more about the Destroy Duchenne Podcast here: https://www.destroyduchenne.org/podcast Website: https://www.destroyduchenne.org

According to reports, there is strong bi-partisan support for House Bill 5321: An Act Requiring Newborn Screening for Duchenne Muscular Dystrophy. If passed, the law would require every newborn in Connecticut to get screened for this starting in July of 2027. We asked Senator Jeff Gordon about the significance of this for families. We also asked about the concerns around hospital consolidations in the state. Sen. Gordon serves on the Public Health Committee and has spent decades of his life as a physician. Image Credit: Getty Images

Jim shares his journey from learning his only son has Duchenne Muscular Dystrophy to running 10 marathons including running over 300 miles from Massachusetts to New Jersey, doing push up around the world and climbing Mt Everest, all to bring awareness to and helping find a cure for this terminal disease. www.jarofhope.org Please subscribe and help keep the Towncast going strong. --- Send in a voice message: https://podcasters.spotify.com/pod/show/flavio-romeo/message

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of givinostat (Duvyzat; Italfarmaco) for the treatment of Duchenne muscular dystrophy (DMD). The therapy, a proprietary histone deacetylase (HDAC) inhibitor, was approved as the first nonsteroidal drug for patients with all genetic variants of DMD. The supporting data for the approval of givinostat comes from the phase 3 EPIDYS trial (NCT02851797). EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Following the approval, Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, sat down to discuss the significance of the approval and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy, its safety profile, and how it may be used with other agents. In addition, she discussed other related topics on gene therapy and unmet needs for this patient population.  For more of NeurologyLive's coverage of givinostat's approval, head here: FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – Givinostat approved for Duchenne muscular dystrophy 2:00 – Sharon Hesterlee, PhD, on the approval's implications 3:35 – Positive downstream effects of the approval 4:50 – Hersterlee on the safety profile of givinostat 5:40 – Promising outlook of the DMD field 6:45 – Overcoming roadblocks involved with gene therapy  8:55 – Remaining unmet needs for patients with Duchenne 10:15 – Closing thoughts on the approval  Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

GDP Script/ Top Stories for Mar 20th    Publish Date:  Mar 19th    From the Ingles Studio Welcome to the Gwinnett Daily Post Podcast. Today is Wednesday, March 20th, and Happy heavenly Birthday to TV host Fred Rodgers. ***03.20.24 – BIRTHDAY – FRED RODGERS*** I'm Bruce Jenkins and here are your top stories presented by Curiosity Lab Criterium. Four Individuals, Including 2 Inmates, Charged with Drug Smuggling in Gwinnett County Jail Georgia Gwinnett College Student Persists in Fight Against Rare Neurological Condition Mountain View Wrestler Vy (Vee) Trinh Honored with Cliff Ramos Perseverance Award After Outstanding Senior Season Plus, my conversation with Leah McGrath from Ingles Markets on Celiac's disease. All of this and more is coming up on the Gwinnett Daily Post podcast, and if you are looking for community news, we encourage you to listen daily and subscribe! Break 1: CURIOSITY LAB BIKE RACE   STORY 1: Four People Accused of Smuggling Drugs Into Gwinnett County Jail Four individuals, including two inmates of the Gwinnett County Jail, have been implicated in a scheme to smuggle drugs into the facility. The operation came to light following the discovery of methamphetamine during a standard cell inspection. The subsequent investigation by the Jail Intelligence Unit led to the uncovering of 10 small packets of methamphetamine, with a total weight of six grams, concealed within the jail. Inmates David Isaias Wilson and Salathiel McKinney are believed to have coordinated the smuggling operation from inside the jail, utilizing external contacts. Willis McFadden, a truck driver, is accused of physically transporting the drugs onto jail premises for McKinney, while Dante Thompson is alleged to have acted as an intermediary, facilitating communication between McFadden and Wilson. The four individuals now face a combined total of 11 felony charges in connection with the drug smuggling operation. STORY 2: 'I'm Not Giving Up' — Georgia Gwinnett College Student Battling Rare Neurological Disease Alejandro Hernandez, 20, diagnosed with Duchenne (Doo-shane) Muscular Dystrophy at age 6, actively confronts the challenges of this incurable disease. Alongside his family, Alejandro has participated in various initiatives to manage DMD, including joining workshops offered by Cure Duchenne. These workshops provide insights into care protocols, therapeutic advancements, and ongoing research, facilitating connections with other families dealing with DMD. Alejandro is pursuing computer programming studies and engages in wheelchair power soccer. He advocates for a positive outlook among DMD patients, emphasizing the importance of maintaining hope. For detailed information on Duchenne Muscular Dystrophy, visit cureduchene.org. STORY 3: Mountain View wrestler Finishes Season By Winning Cliff Ramos Perseverance Award Mountain View High School senior Vy (Vee) Trinh, who has cerebral palsy, demonstrated exceptional perseverance by joining the wrestling team, challenging both medical predictions and personal limitations. Throughout a demanding season, Trinh's commitment remained unwavering, despite achieving limited victories in competition. His resilience and determination garnered respect from both his coach and teammates. At the state championships, Trinh was honored with the FCA Wrestling Coach Cliff Ramos Wrestler of the Year Perseverance Award, recognizing his steadfast spirit. Exhibiting humility and gratitude, Trinh credits his achievements to the support of his team. As he prepares to graduate and pursue studies in cyber security, Trinh's journey stands as a testament to the enduring spirit of determination within the realm of competitive sports. We have opportunities for sponsors to get great engagement on these shows. Call 770.874.3200 for more info. We'll be right back. Break 2: TOM WAGES – GCPS STORY 4: Suit alleging suppression of free speech met with skepticism at U.S. Supreme Court Protesters gathered outside the Supreme Court as it reviewed allegations of government collusion with social media firms to restrict free speech. During the proceedings, justices displayed skepticism towards the lawsuit's foundations. The Biden administration argued against any First Amendment breaches, emphasizing the absence of evidence supporting claims of unconstitutional actions. The lawsuit, brought forward by parties from Missouri and Louisiana alongside individual complainants, suggests that their online expressions were curtailed due to governmental influence. The administration posited that any content moderation was the result of social media platforms enforcing their own policies independently. The justices explored the direct impact of alleged government involvement on the plaintiffs and considered the potential implications for public safety. A decision is anticipated, with the plaintiff's expressing optimism regarding the outcome. STORY 5: Athens Bookshop Files Federal Lawsuit Against Gwinnett Sheriff Avid Bookshop, an independent bookstore located in Athens, has initiated a federal lawsuit against Gwinnett County Sheriff Keybo Taylor and Jail Commander Ben Haynes. The legal action arises from the rejection of books ordered for an inmate, which Avid attributes to a policy that only allows purchases from "authorized retailers," effectively excluding small, local bookstores.   This policy, according to Avid, infringes upon inmates' First Amendment rights and amounts to government censorship. The situation unfolded after Avid Bookshop's attempts to deliver books to an inmate were denied, even though the shipments adhered to the required packaging standards. The Sheriff's Office has defended its policy by highlighting concerns over potential contraband and currently restricts book shipments to those sourced from Barnes and Noble and Amazon.   Avid's legal representatives have criticized the policy as both vague and unconstitutional, arguing it unduly restricts inmates' access to a wider range of reading materials. While the Sheriff's Office maintains that the policy is a measure to ensure safety within the facility, it has chosen not to comment further due to the ongoing litigation. We'll be back in a moment.   Break 3: HENRY CO SHERIFFS   STORY 6: LEAH MCGRATH And now here is my conversation with Leah McGrath from Ingles Markets on celiacs disease. ***LEAH MCGRATH INERVIEW***   We'll have final thoughts after this.   Break 4: INGLES 6 – ATL HEALTH FAIR Signoff – Thanks again for hanging out with us on today's Gwinnett Daily Post podcast. If you enjoy these shows, we encourage you to check out our other offerings, like the Cherokee Tribune Ledger Podcast, the Marietta Daily Journal, the Community Podcast for Rockdale Newton and Morgan Counties, or the Paulding County News Podcast. Read more about all our stories and get other great content at Gwinnettdailypost.com. Did you know over 50% of Americans listen to podcasts weekly? Giving you important news about our community and telling great stories are what we do. Make sure you join us for our next episode and be sure to share this podcast on social media with your friends and family. Add us to your Alexa Flash Briefing or your Google Home Briefing and be sure to like, follow, and subscribe wherever you get your podcasts. Produced by the BG Podcast Network   Show Sponsors: ingles-markets.com wagesfuneralhome.com henrycountysheriffga.gov gcpsk12.org/about-us/careers acc.org/ATLHealthFair peachtreecornersga.gov/385/Curiosity-Lab-Criterium-2024   #NewsPodcast #CurrentEvents #TopHeadlines #BreakingNews #PodcastDiscussion #PodcastNews #InDepthAnalysis #NewsAnalysis #PodcastTrending #WorldNews #LocalNews #GlobalNews #PodcastInsights #NewsBrief #PodcastUpdate #NewsRoundup #WeeklyNews #DailyNews #PodcastInterviews #HotTopics #PodcastOpinions #InvestigativeJournalism #BehindTheHeadlines #PodcastMedia #NewsStories #PodcastReports #JournalismMatters #PodcastPerspectives #NewsCommentary #PodcastListeners #NewsPodcastCommunity #NewsSource #PodcastCuration #WorldAffairs #PodcastUpdates #AudioNews #PodcastJournalism #EmergingStories #NewsFlash #PodcastConversations  See omnystudio.com/listener for privacy information.

Singer songwriter Hannah Connolly, originally from Eau Claire, WI (same as Justin Vernon and the Bon Iver crew!) has just released her second solo album, Shadowboxing. Written to reflect musical and life transitions, it was recorded in beautiful Idyllwild, CA, just outside of her new hometown of Los Angeles. While in the mountain town, Hannah reconnected with nature through hiking and found joy in connecting with her friends and collaborators in music. The process of making the record was crucial for her mental health in music that was celebratory and fun. Her debut album centered around the trauma and healing she and her family faced after her little brother Cullen was killed by a drunk driver in 2015. Born with Duchenne Muscular Dystrophy, Cullen was the life of the party and a bright light in every room he entered. Being able to process and mourn his loss through the making of her first record was not only extremely difficult, but also very necessary for Hannah. We talk about who Cullen was and how he continues to influence Hannah's life and music. These days, Hannah is looking for the fun and lightness again, which is exactly what her little brother would want her to do.Even though Hannah's visual storytelling and folky roots are strong, they are no match for her love of emo music, which has influenced her since she was a teenager. She even performed, recorded and toured in an emo band prior to going solo. Hannah gets into her emo past, her childhood stint in musical theater and, of course, cheese curds. She also gives us the all important update on wedding planning! She recently got engaged to Eric Cannata of the alternative rock band Young the Giant. I'm so happy for Hannah not only for her future marriage, but also for creating this joyful new album. Follow Basic Folk on social media: https://basicfolk.bio.link/ Sign up for Basic Folk's newsletter: https://bit.ly/basicfolknews Help produce Basic Folk by contributing: https://basicfolk.com/donate/ Advertising Inquiries: https://redcircle.com/brands

Gene Therapy's Potential to Transform Rare Disease TreatmentDr. Peter Marks highlights the groundbreaking advancements in gene therapy, especially for rare diseases with high unmet medical needs. He discusses the FDA's role in fostering these innovations, underscoring the importance of understanding gene therapy's application and ensuring its safe delivery. Dr. Marks emphasizes the challenges in manufacturing gene therapies, noting that overcoming these hurdles is essential for treating large populations and common diseases. This insight reflects on the hope and complexity of gene therapy, aiming to provide one-time treatments that could drastically improve patient outcomes.The Role of AI in Enhancing Gene Therapy DevelopmentDr. Marks points out the significant potential use cases for artificial intelligence (AI) in gene therapy, from product design to clinical development and manufacturing. AI's ability to predict off-target effects and streamline the manufacturing process could be a game-changer in this space. By harnessing AI, researchers and developers can optimize safety signals and process large quantities of data for continuous improvement. This insight underscores the potential of AI to revolutionize gene therapy, making it more efficient and effective.Emphasizing the Human Impact of Gene TherapiesDr. Marks and Miller share personal stories of patients and families affected by rare diseases, illustrating the transformative power of gene therapy. They recount profound effects of recent gene therapy approvals on individuals and their communities, particularly highlighting Duchenne Muscular Dystrophy. Marks's narrative conveys the deep emotional and physical impact of gene therapy, not just as a scientific achievement but as a means to significantly enhance lives. This insight brings to light the importance of patient-centered approaches in the development and application of gene therapies.

One of the most rewarding parts of hosting this podcast is connecting with brilliant, compassionate polymaths, whose knowledge and experience extend across numerous realms. Dr. Tanmeet Sethi, MD is a prime example. She is an Integrative and Psychedelic Medicine Physician, activist, TEDx speaker, and author of the incredibly powerful book Joy Is My Justice. She's also been featured on Oprah Daily. Tanmeet has dedicated her career to caring for marginalized patients all over the globe from Seattle to Puerto Rico to Ukraine, as a senior faculty member at The Center for Mind Body Medicine. She is also a Clinical Associate Professor and a primary clinical investigator there on psilocybin at the University of Washington. In this episode, Tanmeet and host Nikhil Torsekar touch on numerous topics, including: The role of movement in releasing myokines / what scientists call hope molecules - whether it's a quiet walk in nature or vigorous dancing to bhangra music. How the vagus nerve regulates our parasympathetic nervous system and helps us feel safe, and how pranayama (or breathwork) facilitates this. The fundamental distinction between happiness (a binary cognitive evaluation) and joy, and how the latter actually comes from the same deep well as pain. An overview of Dr. Sethi's impactful book Joy Is My Justice, which captures her beautiful journey to thrive and embrace with gusto all that life has to offer after her three year old son Zubin received the challenging diagnosis of Duchenne Muscular Dystrophy. Growing up in the South Asian diaspora – the good (solid foundation in community, cultural traditions such as pranayama and Ayurveda), the bad (pressure to live up to impossible standards, tendency to suppress challenging emotions), and the ugly (Nikhil's bhangra moves) To order a copy of Tanmeet's incredible book please visit her Website, or connect with her on Instagram, LinkedIn or Facebook. You can also check out her Substack titled “Beautiful and Brutal.” Connect with Nikhil to learn more about ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Untether Your Life⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ and other projects: ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Instagram⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Website⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠LinkedIn⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠

Listen as Dr. London Smith (.com) and his producer Cameron discuss Duchenne Muscular Dystrophy with special guest Sheila Dawn (Cassie Walker).  Not so boring! https://calderalab.com/pages/podcast-special-offer?show=Jock+Doc&utm_medium=podcast&utm_source=JocDoc https://www.patreon.com/join/jockdocpodcast Hosts: London Smith, Cameron Clark. Guest: Cassie Walker. Produced by: Dylan Walker Created by: London Smith

When he founded Cure Rare Disease in 2018, Rich Horgan was primarily focused on finding a treatment for his younger brother Terry, who had an ultra-rare form of Duchenne Muscular Dystrophy (DMD).   Suffering from a disease that has no cure, Terry served as a “selfless, brave, and courageous” figure that Rich continues to draw inspiration from when seeking new, innovative solutions to treat this progressive, neurodegenerative condition.   Join us for this interview as Rich discusses the origins of Cure Rare Disease, Terry's ongoing legacy, the collaborative effort it takes to advance the drug development journey, the important role of the patient voice when developing treatments, and the future of rare disease research.  Show Notes What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists Letter From Terry's Parents The Disruptors - Meet Rich In Vitro Assays for Duchenne Muscular Dystrophy 2022 World Congress Video Duchenne Muscular Dystrophy Studies Rare Disease Research for Drug Development Scientific Collaboration Leads to Faster Drug Development Cure Rare Disease   

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.##News 1: New treatment for Alzheimer's diseaseIn a groundbreaking development, researchers have announced a new treatment for Alzheimer's disease. The drug, called ADX-100, has shown promising results in early clinical trials. It works by targeting the underlying causes of the disease, rather than just managing the symptoms. This could be a game-changer for the millions of people affected by Alzheimer's worldwide.##News 2: Breakthrough in cancer researchScientists have made a significant breakthrough in cancer research. A new study has identified a genetic mutation that plays a key role in the development of certain types of cancer. This discovery could lead to more targeted treatments and improved outcomes for patients. It is an exciting development in the fight against cancer.##News 3: COVID-19 vaccine updateIn the latest update on COVID-19 vaccines, researchers have found that the Pfizer-BioNTech and Moderna vaccines provide strong protection against the Delta variant of the virus. This is reassuring news as the Delta variant continues to spread globally. Vaccines remain our best defense against the virus, and these findings further support their efficacy.##News 4: Gene therapy shows promise for rare genetic disorderGene therapy has shown promise in treating a rare genetic disorder known as Duchenne muscular dystrophy. In a recent clinical trial, patients who received the gene therapy experienced significant improvements in muscle strength and function. This is an encouraging development for those living with this debilitating condition.##News 5: Advances in personalized medicineAdvances in personalized medicine are revolutionizing healthcare. Researchers are now able to analyze an individual's genetic makeup to tailor treatments specifically to their needs. This approach has shown great promise in treating conditions such as cancer, where targeted therapies can be more effective and result in fewer side effects.##News 6: New drug for diabetes managementA new drug has been approved for the management of diabetes. This medication, called GlucoControl, helps regulate blood sugar levels and has shown to be effective in controlling the disease. With the number of people with diabetes on the rise, this new treatment option is a welcome addition to the available therapies.##News 7: Advances in gene editing technologyGene editing technology has made significant advancements in recent years. Scientists can now make precise changes to an organism's DNA, opening up possibilities for treating genetic diseases and improving agricultural practices. This technology has the potential to revolutionize various industries and improve human health.##News 8: Promising results in HIV vaccine trialA new HIV vaccine has shown promising results in a recent clinical trial. The vaccine, which uses a novel approach to stimulate the immune system, demonstrated a high level of efficacy in preventing HIV infection. This is a major breakthrough in the search for an effective HIV vaccine and brings hope for the future.##News 9: Artificial intelligence in drug discoveryArtificial intelligence (AI) is transforming the field of drug discovery. Machine learning algorithms can analyze vast amounts of data to identify potential drug candidates more efficiently than traditional methods. This has the potential to accelerate the development of new treatments and improve patient outcomes.##News 10: Progress in regenerative medicineRegenerative medicine holds great promise for treating a wide range of diseases and injuries. Scientists are making significant progress in growing organs and tissues in the lab, as well as developing therapies to stimulate the body's natural healing processes. This field has the potential to revolutionize healthcare in the coming years.That's all for today's episode. Stay tuned

Ken Mills, CEO, President, and Director at REGENXBIO joins Erin Harris for this episode of Cell & Gene: The Podcast to discuss the company's progress with its Phase I/II Affinity Duchenne trial of RGX-202 for the treatment of Duchenne Muscular Dystrophy. They discuss the future of AAV gene therapy as well as achieving scalability with consistent yield and product purity in gene therapy manufacturing.

Hour 3 starts with something much more important than sports. Rachel Poysky, from Coach to Cure (www.coachtocureMD.org), joined John & Del to discuss Duchenne Muscular Dystrophy, how it effects our young men, and how you can help the cause to find cure by donating. How will C.J look against that Steelers defensive front? Tonight is a must win for the Astros in Seattle. The show wraps with a recap of what is needed for the Stros to backpedal into the Post-season, and News of the weird.

To have your question featured in a future video, please email: questions@drmdc.health

Christopher Davison has faced many difficulties because of his Duchenne muscular dystrophy, a progressive, terminal illness that has led to quadriplegia. Diagnosed at age 6 and in a wheelchair by 8, Christopher didn't expect to live past his 20s. Now in his 30s, he lives to serve God and help others to find hope and truth in their difficulties. He joins the podcast to talk about living with a progressive disability, his experience as a pastor for 10 years, and how God's strength shines through brightest in our own weakness.Christopher earned a Bachelor of Science degree in Ministry Leadership from Moody Bible Institute and has his Master of Divinity degree from Carolina University. After serving for 10 years as an associate pastor, he founded an online ministry, Wheel Truth Media, to help Christians grow in their faith.Watch Christopher's testimony KEY QUESTIONSDo you trust God with your life?How have you seen God's strength shine through your weakness? KEY SCRIPTUREPhilippians 1:20–21: “I eagerly expect and hope that I will in no way be ashamed, but will have sufficient courage so that now as always Christ will be exalted in my body, whether by life or by death. For to me, to live is Christ and to die is gain.” Connect with Chris on: YouTube, Facebook, Twitter, Instagram.---- Find more encouragement on Joni Eareckson Tada's Sharing Hope podcast  and daily devotional.Follow Joni and Friends on TikTok, Instagram, Facebook, and YouTube.Your support  makes this podcast possible!Joni and Friends envisions a world where every person with a disability finds hope, dignity, and their place in the body of Christ. Founded by Joni Eareckson Tada, we provide Christ-centered care through  Joni's House, Wheels for the World, and Retreats and Getaways, and offer disability ministry training.

Chris Jones and his wife Natalie live in Salt Lake City, Utah, and are the parents of 4 children and grandparents of two. Chris is a fiercely creative entrepreneur who builds businesses and products that build people. He's a prolific writer whose work has been widely recognized as unflinchingly authentic, insightful, and an invitation to live a more examined life. Chris's work has since gained international attention as a place to journey into the often-misunderstood world of grief, healing, and the pursuit of meaning My interview with Chris today is all about grief, healing and trauma, something nobody wants to be an expert in from experience, but he's been through the trenches with after losing his 10 year old son Mitchell to Duchenne Muscular Dystrophy in 2013. Chris wrote, "Among the recurring themes of Mitchell's Journey are discussions of faith, making sense of sorrow, and reflections on love and loss. I suppose one could add to those themes the singularity of grief, that after all is said and done, the journey of grief is traveled by one." His perspective on loss and suffering is one that touched me deeply when Neil and I met him in Hawaii last year at a personal development retreat. We also talk about Chris's work called A Thousand Points of Light - the idea that we all have points of light in our lives, even in our darkest moments, and they often intersect with the people around us without us even knowing. I always learn so much from Chris and I'm really excited and grateful to share his wisdom and life experiences with you today too. Time Stamps: [01:41] - Chris Jones speaks about his son Mitchell and having a reoccurring impression that something is coming. [07:38] - What did Chris do to start his healing journey? [12:08] - How can someone mourn with others who mourn? [15:57] - What is A Thousand Points of Light? [24:35] - Chris shares his view on learning from trauma and how he redefines what role trauma plays in life.  [30:53] - How can we carry grief as time moves on? [34:30] - “There are things we can do to exercise our minds and our hearts to become stronger.” [37:38] - Are you being kind to yourself? Are you being kind to others? Supporting Resources: Facebook: Mitchell's Journey  Mitchell's Journey website  A Thousand Points of Light website  "A Thousand Points of Light: In Search of Tender Mercies" Video Learn more about your ad choices. Visit megaphone.fm/adchoices