Podcasts about sarepta therapeutics

Der Bundestag gibt grünes Licht für das Billionenpaket - der DAX steigt daraufhin um 1,0 % auf 23.381 Punkte und erreicht zuvor ein neues Rekordhoch bei 23.476,01 Punkten. EuroStoxx50 +0,7 % bei 5482 Punkten. Euro stabil bei 1,0934 US-Dollar. Siemens baut weltweit 6.000 Stellen ab, 2.850 in Deutschland, vor allem in der Sparte Digital Industries. RWE sichert sich einen 15-jährigen Solarstrom-Vertrag mit Meta für einen 200-Megawatt-Park in Texas. Xiaomi übertrifft Erwartungen mit +69 % Nettogewinn (1,05 Mrd. Euro) und Umsatzplus von fast 50 %. Sarepta Therapeutics verliert vorbörslich knapp 25 %, nach Tod eines Gentherapie-Patienten. Nvidia gibt vor KI-Rede von CEO Huang um 2 % nach. Steyr Motors setzt Höhenflug nach Rheinmetall-Deal fort. Börsenweisheit: "Kaufen, wenn die Kanonen donnern, verkaufen, wenn die Violinen spielen." - Carl Mayer von Rothschild. In den Interviews: Carsten Klude: Deutschland wird der großzügige Onkel, der viel Geld in die Hand nimmt. "Rüstung ist vielleicht die neue KI". Heiko Thieme: "Trump schadet Amerika!" - Börsen, China, Tech-Aktien und Strategien.

In this episode, mechanisms and research are discussed. The question, ‘if we have a randomized control trial (RCT), can we do without knowledge of a mechanism?' is answered. It is held that mechanisms do make a substantive difference to the optimization of a RCT. This is defended through two cases — the failed Duchenne Muscular Dystrophy Phase 3 trials in 2024 ran by Sarepta Therapeutics and Pfizer and the daptomycin 2005 trial to test its efficacy in patients Gram-positive community acquired pneumonia. Nancy Cartwright's INUS contributors are put forth as a potential objection. However, an as-complete-as-possible concept of mechanistic understanding and reasoning is advocated for ultimately.

In this episode we discuss the recent National Society of Genetic Counselor's Practice Resource on Dystrophinopathies, which was recently published in JoGC, with two of the publications' authors. You can find the Journal of Genetic Counseling webpage via onlinelibrary.wiley.com or via the National Society of Genetic Counselors website.   Segment 1: “Genetic counseling for the dystrophinopathies- Practice resource of the National Society of Genetic Counselors”   Ann Martin, MS, CGC is a board certified genetic counselor with Parent Project Muscular Dystrophy (PPMD). Ann serves as the VP of Community Research and Genetic Services and manages a team of genetic counselors who coordinate all aspects of The Duchenne Registry and the Decode Duchenne genetic testing program. Since joining PPMD in 2011, Ann has been directly involved in all aspects of the Registry programs including collection and curation of the Registry data, clinical trial and research study recruitment, data exports, patient and family inquiries, genetic testing, and educational content on the PPMD websites. Ann represents The Duchenne Registry on TREAT-NMD's Global Data Oversight Committee, which is responsible for reviewing requests for data from the TREAT-NMD Global Registries, and serves as the DMD Subgroup Co-Lead. In addition, Ann has been a member of Sarepta Therapeutics' Advisory Board for Early Diagnosis of Duchenne since 2022. Ann is continually engaging and educating both the patient and professional community about the Registry and Duchenne and Becker research. Before joining PPMD, Ann worked as a clinical genetic counselor for 15 years at Carolinas Medical Center in Charlotte, NC, where she provided genetic counseling for pediatric and adult patients with a wide range of genetic disorders. Ann is a graduate of the University of Cincinnati Genetic Counseling Graduate Program. She is board-certified by the American Board of Genetic Counseling and is a member of the National Society of Genetic Counselors. Angela Pickart (she/her) is a licensed, certified genetic counselor practicing in the Genomics Laboratory at Mayo Clinic, performing variant interpretation, report writing and test development for neurogenetics assays. She received her Master of Science degree in Genetic Counseling at the University of Minnesota and has extensive clinical experience in pediatric, neurology, and oncology genetic counseling prior to transitioning to her laboratory role. She has been awarded the academic rank of Assistant Professor of Laboratory Medicine at the Mayo Clinic College of Medicine and Science where she serves as course co-director of the Center for Clinical and Translational Sciences Molecular Variant Evaluation Course. She is also the course co-director of the Laboratory Practicum for the Medical College of Wisconsin Master of Science Genetic Counseling Graduate Program and Adjunct Assistant Professor of the Medical College of Wisconsin School of Graduate Studies.   In this segment we discuss: - Origins and significance of the genetic counseling-specific practice resource for dystrophinopathies. - Changes in the treatment landscape due to FDA-approved therapeutics and ongoing clinical trials for dystrophinopathies. - Strategies for educating patients and families about the implications of genetic testing and diagnosis in dystrophinopathies. - Advocacy roles of genetic counselors in helping patients access the latest therapies and participate in clinical trials for DMD/BMD. - Importance of interdisciplinary collaboration in managing dystrophinopathies and the roles genetic counselors play within these teams. - Potential broader impact of the practice resource on the medical field and patient community.    Would you like to nominate a JoGC article to be featured in the show? If so, please fill out this nomination submission form here. Multiple entries are encouraged including articles where you, your colleagues, or your friends are authors. Stay tuned for the next new episode of DNA Dialogues! In the meantime, listen to all our episodes Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “DNA Dialogues”.  For more information about this episode visit dnadialogues.podbean.com, where you can also stream all episodes of the show. Check out the Journal of Genetic Counseling here for articles featured in this episode and others.  Any questions, episode ideas, guest pitches, or comments can be sent into DNADialoguesPodcast@gmail.com.  DNA Dialogues' team includes Jehannine Austin, Naomi Wagner, Khalida Liaquat, Kate Wilson and DNA Today's Kira Dineen. Our logo was designed by Ashlyn Enokian. Our current intern is Sydney Arlen.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Lilly emerged victorious in a head-to-head weight loss battle against NovoPlus, showcasing the effectiveness of tirzepatide over semaglutide. This victory has highlighted the potential of tirzepatide in promoting weight loss, with results aligning with real-world studies. Meanwhile, Amylin has garnered attention in the weight loss sphere ahead of Novo's cagrisema readout, with industry anticipation for the phase III results. Amylin analogs are seen as a promising alternative to GLP-1 receptor agonists, offering potentially higher-quality weight loss with improved tolerability. Additionally, GSK has turned to China for another potential ADC deal worth over $1 billion with Dualitybio.The Trump administration's impact on the biopharma industry is expected to be mixed, with potential promotion of M&A activity but concerns about vaccine sales due to controversial picks. Intra-Cellular is seeking FDA approval for Caplyta in major depressive disorder, which could generate significant sales. Sarepta Therapeutics, known for regulatory success, has made a significant deal with Arrowhead Pharmaceuticals for RNA-based medicines. Other news includes Lilly's victory over Novo in a weight loss study, anticipation for Novo's phase III Cagrisema readout, Novartis' deal with PTC for Huntington's disease, and more. Takeda is investing in Keros' blood cancer anemia drug, Roche partners with Cour in an autoimmune deal, and Gilead is planning an ADC comeback with a potential license agreement. Alligator Bioscience has cut staff due to capital constraints.

Genethon, the non-profit gene therapy developer created by the patient association AFM-Telethon, began working with Sarepta Therapeutics in 2017 to develop a gene therapy for the rare neuromuscular condition Duchenne muscular dystrophy. Now that Sarepta has won approval for a separate gene therapy to treat the condition, Genethon is advancing development of its experimental gene therapy on its own. We spoke to Frederic Revah, CEO of Genethon, about Duchenne, the organization's efforts to complete clinical development of its gene therapy for the condition, and how it might commercialize the treatment.

In this episode of Molecule to Market, you'll go inside the outsourcing space of the global drug development sector with Chris Garabedian, CEO at Xontogeny and Venture Fund Manager at Perceptive Advisors. Your host, Raman Sehgal, discusses the pharmaceutical and biotechnology supply chain with Chris, covering: How he utilized his pharma experience to become a CEO and revitalize a biotech company, positioning it for a $15bn market cap Why the biotech space had such a market downturn, and how the uptake in the industry will be data-driven Demystifying the role of a biotech CEO and understanding what it takes to drive successful drug development Why relying on outsourcing is critical for biotechs today The emerging, secondary-tier biotech clusters in the US... but why does Boston remain unrivalled? Chris Garabedian founded Xontogeny in June of 2016 to support multiple promising technologies from early development through clinical proof of concept. In 2017, Chris joined Perceptive Advisors to develop their Venture Fund strategy and is Portfolio Manager of the Perceptive Xontogeny Ventures Fund, which supports early-stage companies with Series A investments across biotech, medtech and healthtech. Chris has a broad base of experience and a track record of success over his decades-long experience in the biopharma industry. Chris served as the President and CEO of Sarepta Therapeutics from 2011 to 2015, overseeing the turnaround of a company that is now a commercial-stage leader in the genetic technology space. He also led Corporate Strategy for Celgene from 2007 to 2010 and served in several global commercial and corporate development leadership roles at Gilead from 1997 to 2005. He serves on the boards of several life sciences companies and speaks at industry conferences on a wide range of important issues. He is a member of the Corporate Relations Board for the Keck Graduate Institute. He has previously served on the Board of Directors of MassBio and is a Senior Advisor for the Boston Consulting Group. ----- Join Molecule to Market and ramarketing for an exclusive panel discussion series at this year's CPHI Milan - across 3 power-packed sessions, our industry experts will be sharing insights into driving brand growth with strategic marketing. Find out more and pre-register here ----- Please subscribe, tell your industry colleagues and join us in celebrating and promoting the value and importance of the global life science outsourcing space. We'd also appreciate a positive rating! Molecule to Market is sponsored and funded by ramarketing, an international marketing, design, digital and content agency helping companies differentiate, get noticed and grow in life sciences.

What would it be like to dedicate over 50 years of your life towards treating a disease? Today, we're finding out with Dr. Jerry Mendell, who has been working to help patients with Duchenne muscular dystrophy since the 1960s. He is known for studying a similar disorder and partnering with Sarepta Therapeutics to create a therapy that delivers healthy copies of the dystrophin gene, which was approved by the FDA in June of 2023. Join us to hear Dr. Mendell's thoughts on applying new forms of gene therapy for this condition, extrapolating his findings to treat spinal muscular atrophy, and the onslaught of modern research becoming outdated in the context of accelerating technological developments.

We love to hear from our listeners. Send us a message.Debra Miller, CEO and Founder and Dr. Michael Kelly, Chief Scientific Officer at CureDuchenne talk to Host Erin Harris about the FDA approval for the expansion to the label indication for ELEVIDYS from Sarepta Therapeutics as well as how CureDuchenne works with various biotechs developing gene therapies for Duchenne muscular dystrophy (DMD). Miller and Kelly also share their advice and recommendations for companies developing drugs for DMD - while much progress has been made, more innovation is needed.

Aktien hören ist gut. Aktien kaufen ist besser. Bei unserem Partner Scalable Capital geht's unbegrenzt per Trading-Flatrate oder regelmäßig per Sparplan. Alle weiteren Infos gibt's hier: scalable.capital/oaws. Aktien + Whatsapp = Hier anmelden. Lieber als Newsletter? Geht auch.  Das Buch zum Podcast? Jetzt lesen.  NVIDIA verliert ein SAP, Apple verliert Hoffnung, Carlsberg verliert bei Übernahme von Britvic. Außerdem nimmt Boeing seine Probleme selbst in Besitz, Sarepta Therapeutics nimmt für 1 Gentherapie 3 Millionen, Asana kauft Aktien, Zealand verkauft Diät-Pillen. Deutschland reguliert, aber wer profitiert? Vielleicht freenet (WKN: A0Z2ZZ). Was NVIDIA für Chips ist, ist Bachem (WKN: A3DLKE) für Abnehmspritzen. Wir erklären, wieso und eine Runde Bio-LK gibt's gratis on top. Diesen Podcast vom 24.06.2024, 3:00 Uhr stellt dir die Podstars GmbH (Noah Leidinger) zur Verfügung.

Plus: Shares in several major banks fall after federal regulators raise concerns about their wind-down plans. Shares in Sarepta Therapeutics jump after its new muscular dystrophy gene therapy receives approval. And Nikola's stock rises after the embattled EV maker's board approves a reverse stock split. Danny Lewis hosts. Sign up for the WSJ's free What's News newsletter. Learn more about your ad choices. Visit megaphone.fm/adchoices

The Nasdaq and S&P 500 notched their third straight positive weeks; Unlimited Funds' Bob Elliott and Cantor Fitzgerald's Eric Johnston break down their top trades. Olaulu Aganga, Mercer US CIO, on the playbook for the second half. Ben Reitzes, Melius analyst, on why he's upping his Nvidia price target again, despite the stock's decline this week. TD Cowen's Riu Baral on Sarepta Therapeutics' huge move on FDA's expanded use approval for its DMD drug. Plus, a cyberattack is forcing car dealerships back to old-fashioned pen and paper. 

Die Wall Street hat gestern ein anderes Bild als an den meisten Tagen zuvor gezeigt. Diesmal waren die Techwerte die Verlierer. Old Economy wiederum präsentierte sich stark. Heute ist erst einmal Richtungssuche angesagt.

In der heutigen Folge „Alles auf Aktien“ sprechen die Finanzjournalisten Anja Ettel und Holger Zschäpitz über einen Terrorabschlag bei Tesla, die Teppich-Enttäuschung bei Bayer und aussichtsreiche M&A-Kandidaten. Außerdem geht es um Fresenius Medical Care, Novo Nordisk, Nio, Apple, Target, Walmart, Lufthansa, Fresenius Medical Care, LEG Immobilien, ThyssenKrupp, Fonds DJE - Mittelstand & Innovation (WKN: A14SK0), Berenberg Aktien Mittelstand (WKN: A2JF7M), DWS Concept Platow (WKN: DWSK62), Exxon Mobil, Bristol Myers Squibb, AT&T, Zealand Pharma, Viking Therapeutics, Tenet Healthcare, Sarepta Therapeutics, Tripadvisor, Wingstop, Victoria's Secret, Global Holdings, Viatris, Multi Axxion Europa Fonds (WKN: 765377), Invesco EQQQ Nasdaq-100 (WKN: 801498) und WisdomTree NASDAQ 100 3x Daily Leveraged (WKN: A3GL7E). Wir freuen uns an Feedback über aaa@welt.de. Ab sofort gibt es noch mehr "Alles auf Aktien" bei WELTplus und Apple Podcasts – inklusive aller Artikel der Hosts und AAA-Newsletter. Hier bei WELT: https://www.welt.de/podcasts/alles-auf-aktien/plus247399208/Boersen-Podcast-AAA-Bonus-Folgen-Jede-Woche-noch-mehr-Antworten-auf-Eure-Boersen-Fragen.html. Disclaimer: Die im Podcast besprochenen Aktien und Fonds stellen keine spezifischen Kauf- oder Anlage-Empfehlungen dar. Die Moderatoren und der Verlag haften nicht für etwaige Verluste, die aufgrund der Umsetzung der Gedanken oder Ideen entstehen. Hörtipps: Für alle, die noch mehr wissen wollen: Holger Zschäpitz können Sie jede Woche im Finanz- und Wirtschaftspodcast "Deffner&Zschäpitz" hören. Außerdem bei WELT: Im werktäglichen Podcast „Das bringt der Tag“ geben wir Ihnen im Gespräch mit WELT-Experten die wichtigsten Hintergrundinformationen zu einem politischen Top-Thema des Tages. +++ Werbung +++ Du möchtest mehr über unsere Werbepartner erfahren? Hier findest du alle Infos & Rabatte! https://linktr.ee/alles_auf_aktien Impressum: https://www.welt.de/services/article7893735/Impressum.html Datenschutz: https://www.welt.de/services/article157550705/Datenschutzerklaerung-WELT-DIGITAL.html

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Stephen From, chief executive officer of Aruna Bio, sat down to discuss the company's investigational exosome candidate AB126 and its upcoming phase 1b/2a trial in patients with ischemic stroke. He discussed the mechanism of action of the therapy, the advantages of its anti-inflammatory and neuroprotective properties, and how it will be used with other stroke treatments. Furthermore, he gave insight on the complexities with conducting stroke trials and improving neurodegeneration in poststroke patients.  Looking for more stroke discussion? Check out the NeurologyLive® stroke clinical focus page. Episode Breakdown: 1:00 – Overviewing phase 1b/2a trial 7:05 – Mechanism of action of AB126 11:15 – Neurology News Minute 13:45 – Role of AB126 in stroke treatment landscape 17:50 – Challenges with repairing neurodegeneration in stroke This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: European Commission Approves Omaveloxolone as First Therapy for Friedreich Ataxia AVP-786 Falls Short in Phase 3 Study of Alzheimer Disease Agitation FDA Grants Priority Review to Efficacy Supplement for Sarepta Therapeutics' SRP-9001 Indication Expansion 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.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Sarepta Therapeutics has released data on its new drug for Duchenne muscular dystrophy, showing potent boosting of dystrophin protein production. However, some side effects, including electrolyte imbalances, were also highlighted. Astrazeneca and Daiichi are seeking FDA approval for their drug Enhertu as a "tumor agnostic" treatment, and the FDA is currently reviewing their application. Bristol Myers has received positive data from a trial of a subcutaneous formulation of Opdivo in kidney cancer, showing similar efficacy to the intravenous formulation. Biotech IPOs continue to be an important source of funding for the industry, with Alto Neuroscience and Fractyl Health preparing for initial stock offerings. Merck Manuals is focusing on a "one health" approach, integrating human, animal, and environmental health in medical education and practice.Philips has announced that it will stop selling sleep and respiratory devices in the US due to a consent decree from the FDA. This decision comes as Philips faces regulatory scrutiny and potential legal action related to quality issues with its sleep apnea devices. Medical device companies Stryker, Boston Scientific, and BD are set to post their earnings this week. Absolutions has received FDA breakthrough designation for its abdominal wall closure device, aiming to reduce the risk of hernia by distributing suture tension over a large area of tissue. Globus has laid off employees from NuVasive following their merger. Masimo has forced Apple to redesign its smartwatches in a long-running patent dispute.The pharmaceutical industry is in a race to develop groundbreaking drugs for pain relief. Tanezumab, developed by Eli Lilly and Pfizer, was dropped due to safety concerns. Attention has turned to Vertex Pharmaceuticals' candidate vx-548, which has shown positive results in phase 2 trials for chronic nerve pain. South Rampart Pharma has won fast track designation for its novel drug that activates pain signaling pathways without causing liver and kidney toxicities. Tonix Pharmaceuticals is repurposing an existing technology for the treatment of fibromyalgia, with positive late-stage results. These developments offer hope for patients suffering from chronic pain conditions and could generate significant revenue for the companies involved.Overall, these developments highlight ongoing challenges and changes in the medtech industry, including regulatory scrutiny, mergers, and patent disputes. Medtech companies must navigate these complexities while leveraging connected ecosystems, data, and strategic planning to drive innovation in healthcare.Thank you for tuning in to Pharma and Biotech daily. Stay informed, stay ahead.

Zajímá vás, co vedlo k více jak 45% propadu  Sarepta Therapeutics (SRPT) 31. října? A je teď vhodná příležitost této akcie k nákupu?  Poslechněte si krátký rozhovor s Jánem Hájkem z Top Stocks, který jsme natočili na České investiční konferenci. ZPĚTNÁ VAZBA:Vaše zpětná vazba je pro mě důležitá. Máte-li k podcastu jakékoliv poznámky, zašlete mi je, prosím, na lenka@schanova.com. Děkuji. 

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Today, we have some interesting news to share with you. Let's get started.## Artificial Intelligence Revolutionizing Regulatory Approval ProcessesArtificial intelligence (AI) is making advancements in regulatory approval processes in the life sciences industry. It has the potential to revolutionize these processes by accelerating and improving consistency and confidence. AI can significantly speed up the regulatory approval process, which currently takes an average of 10 years for drug development. Embracing new technologies like AI is crucial for the industry to move forward.## Abzena: A Leader in Biologics and BioconjugatesAbzena, a contract development and manufacturing organization (CDMO), specializes in biologics and bioconjugates. They have a proven track record of helping clients discover lead candidates, create production cell lines, and deliver programs for investigational new drug (IND) applications. Their effectiveness at getting things done has led to 85% of clients returning to work with them again.## Sarepta Therapeutics Shares Drop After Failed StudySarepta Therapeutics' shares dropped by more than 40% after the results of the Embark study failed to meet its main goal. The study aimed to confirm the approval of its gene therapy Elevidys for treating Duchenne muscular dystrophy. However, there is still hope for gene editing technology as the FDA reviews Vertex Pharmaceuticals and CRISPR Therapeutics' case for approval of a sickle cell therapy.## Eli Lilly Acquires Beam TherapeuticsEli Lilly has acquired Beam Therapeutics in a deal worth $200 million upfront and an additional $50 million investment. This acquisition gives Lilly option rights to several gene editing programs. It is an exciting move that showcases the company's commitment to advancements in gene therapy.## Pfizer's RSV Vaccine Sees Strong SalesPfizer's respiratory syncytial virus (RSV) vaccine, Abrysvo, has seen over $300 million in sales during its first few months on the market. This success is expected to help offset declining sales of COVID-19 vaccines. It is a positive development in the fight against respiratory diseases.## Promising Results for Novartis' Kidney Disease DrugNovartis' bet on a kidney disease drug has yielded positive results in a phase 3 trial. The therapy, acquired through the acquisition of Chinook Therapeutics, met its goals in the trial. This success highlights the potential for advancements in treating kidney diseases.## Neuroscience Drug Development Shows PromiseIn the field of neuroscience drug development, there are promising new drugs for conditions such as Alzheimer's, ALS, and depression. These developments show that neuroscience is becoming a priority again in the biopharmaceutical industry. It is an exciting time for advancements in treating neurological disorders.## Philips Recalls Sleep Apnea Devices, GE Healthcare Remains ResilientPhilips has issued multiple recalls of its sleep apnea devices and ventilators due to problems with soundproofing foam. Despite these recalls, GE Healthcare's sales have remained resilient. The company has raised its earnings forecast for 2023 and plans to expand margins. They are navigating challenges such as China's anti-corruption campaign.## Amgen Takes Write-down, Sarepta Fails Phase III TrialAmgen has announced a $650 million write-down after discontinuing its prostate cancer drug candidate. Sarepta Therapeutics has also failed to meet the primary endpoint in a Phase III trial of its gene therapy for Duchenne

Duchenne muscular dystrophy is a devastating disease and, until very recently, was one without much hope. When Hawken Miller was diagnosed at age 5, the physician told his parents to enjoy the time they had with him, as there wouldn't be much. Over 20 years later, Miller is a journalist and content strategist for CureDuchenne, an organization started by his mother when he was diagnosed, and new treatment is on the horizon. Last month, the FDA approved a new gene therapy from Sarepta Therapeutics that will provide hope for many patients and families. The new drug, however, has its limitations — it's only approved for children age 4-5, for example. Miller joined the podcast to discuss how this drug may change lives, what more needs to happen, and what his personal experience has been living with Duchenne.

Full approval of Leqembi lecanemab-irmb will unlock reimbursement and energize Alzheimer's R&D, but the launch will be slow, said BioCentury Washington Editor Steve Usdin on the latest BioCentury This Week podcast. Usdin and colleagues break down the takeaways from BioCentury's interview with Chris Viehbacher, CEO of Biogen, and discuss the challenges of launching a product for a disease that has until now been untreatable.BioCentury's editors also discussed the prospects for the IPO window in the second half now that the market for follow-on financings is showing signs of life, and the controversy surrounding Elevidys delandistrogene moxeparvovec, the gene therapy from Sarepta Therapeutics for Duchenne muscular dystrophy that Usdin argued in an Editor's Commentary shows flaws in evidence generation and regulatory processes.

In the latest installment of Ropes & Gray's Alumni @ RopesTalk podcast, life sciences partner Amanda Austin, who's also a member of the firm's alumni committee, interviews alum and friend Heidy (Abreu) King-Jones, former chief legal officer at Provention Bio. Heidy was an associate at Ropes & Gray from 2008 – 2013, after which she took in-house roles at Sarepta Therapeutics, Axcella and Provention Bio. As a self-described “student of law, student of biotech and student of life,” Heidy talks broadly about her career, with detours into the experience of working at Provention Bio during its acquisition by Sanofi. She shares what she learned at Ropes & Gray, including an important lesson about client service. For anyone thinking about next steps in their career, Heidy also offers great advice focused on where you find professional joy versus professional drain. This interview was recorded on May 25, shortly before Heidy's departure from Provention Bio, which was acquired by Sanofi in April.

FDA Commissioner Rob Califf's biggest legacy could be his reform of FDA's advisory committee process. But what is uncertain is whether his review can bring together enough consensus among agency leaders to drive a meaningful change, said Washington Editor Steve Usdin on the latest BioCentury This Week podcast. Usdin and colleagues discuss the areas of the U.S. regulator's advisory committee process where there is — and isn't — agreement on needed changes, and what could make this time different from a lengthy history of calls for change. BioCentury's editors also preview an upcoming discussion with Polaris Partners' Amy Schulman regarding why Singapore, the funding scene, and women in leadership. Usdin then discusses why reimbursing the recently approved DMD gene therapy from Sarepta Therapeutics through state Medicaid plans won't be so simple.

Get our free download! Implementing AMA Style – 8 Things to Get Right In this episode, we discuss the latest FDA approvals from June 19 –  June 23, 2023.  Here are the key highlights: ·       The FDA has given approval to Pfizer's Talzenna and Xtandi for treating metastatic castration-resistant prostate cancer in adults with certain genetic mutations. The approval came following successful Phase 3 TALAPRO-2 trial results. ·       Sarepta Therapeutics' Elevidys, a gene therapy for Duchenne muscular dystrophy, has received accelerated approval from the FDA. Elevidys, the first gene therapy of its kind, is approved for ambulatory pediatric patients aged 4-5 years with confirmed mutation in the DMD gene. ·       Jardiance and Synjardy, initially approved for adults in 2014 and 2015 respectively, have now been approved by the FDA for use in children aged 10 and older with type 2 diabetes. This approval came on the back of the DINAMO phase 3 trial results. ·       Pfizer's Litfulo, a once-daily oral treatment for severe alopecia areata for individuals aged 12 and older, has received FDA approval. This is the first treatment approved for adolescents with this condition.  ·       Amgen's Blincyto has received FDA approval for the treatment of CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL) in first or second complete remission with minimal residual disease. This moves the drug from an accelerated approval to a full approval status. ·       Argenx's Vyvgart Hytrulo, a subcutaneous version of the original drug Vyvgart, has been approved by the FDA. It is indicated for generalized myasthenia gravis (gMG) in adult patients who test positive for anti-acetylcholine receptor antibodies. ·       Bayer's Ultravist (iopromide) injection, the first contrast agent for contrast-enhanced mammography, has been approved by the FDA. This new imaging tool enhances visibility of suspected or known breast lesions. ·       The FDA has refused to grant accelerated approval to Intercept Pharmaceuticals' drug obeticholic acid (OCA) for treating non-alcoholic steatohepatitis (NASH). This is the second time the FDA has declined to approve the drug for NASH due to potential risks. ·       Please check back every Monday morning for last week's approvals so that you can stay up to date.  See all the episodes here. ·       This podcast is brought to you by Nascent Medical. If you're a project manager at a CME or medical communications agency and need on-call medical writing assistance please visit Nascent Medical. We are a team of MD- and PhD-level medical writers and can create slide decks, white papers, ad board summaries, manuscripts, needs assessments, and much more. We also do medical editing using AMA style and factchecking. Visit nascentmc.com  ·       Intro and outro music ·       Garden Of Love by Pk jazz Collective

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 SRP-9001, or delandistrogene moxeparvovec (Elevidys; Sarepta) in Duchenne muscular dystrophy (DMD). Offering commentary is Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis. SRP-9001 is an AAV vector-based gene therapy that was approved for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene aged 4 to 5 years, based on data from the phase 3 EMBARK study (NCT05096221). For more of NeurologyLive®'s coverage of SRP-9001's approval, head here: FDA Approves SRP-9001 as First Gene Therapy for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – SRP-9001 (Elevidys; Sarepta) is approved for DMD 1:35 – Natalie Goedeker, CPNP, on the approval 3:05 – Efficacy of SRP-9001 4:00 – Goedeker on pieces of efficacy data released 4:45 – Currently available therapies for DMD 5:35 – Goedeker on role of SRP-9001 in treatment landscape 6:55 – Safety data of SRP-9001 8:00 – Goedeker on safety considerations of which prescribing clinicians should be aware 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. REFERENCES 1. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. News release. June 22, 2023. Accessed June 22, 2023. https://www.businesswire.com/news/home/20230622454844/en/

In this episode, Briana Contreras, an editor with Managed Healthcare Executive, and Peter Wehrwein, managing editor of MHE, got to chatting with the newest editorial advisory board member, Jeffrey Casberg, who is vice president of Clinical Pharmacy at IPD Analytics.  In the discussion, Casberg shared the most pressing or prominent issues on his mind, he explained what the drug life-cycle is at IPD, and what the latest is on drugs in the pipeline. Casberg also shared his predictions for Sarepta Therapeutics' DMD drug for young children and Mounjaro's Type 2 Diabetes Drug, and even touched on some common misconceptions he notices about drug approvals, pricing and cost.

Synopsis: Kate Haviland is the CEO of Blueprint Medicines, a global precision therapy company that creates life-changing medicines for people with cancer and blood disorders. Applying an approach that is both precise and agile, they create medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Kate discusses beginning her career at Genzyme before joining Blueprint Medicines, and what the transition to becoming CEO was like after having held various different roles at Blueprint. She also talks about how personalized medicine is transforming patient care, and the company's focus on applying precision medicine to cancer and hematologic diseases. Finally, she dives into how the current environment informs how she approaches leading a publicly traded biotech and her perspective on partnerships. Interested in learning more? Check out their recent approval here: https://rb.gy/4126e Biography: Kate brings an impressive breadth of experience and leadership within the biopharmaceutical industry, as well as a substantial background in business development, commercial and strategic planning, and program management. Since April 2022, Kate has served as Chief Executive Officer of Blueprint Medicines. Kate joined Blueprint in 2016 as Chief Business Officer and later served as Chief Operating Officer, where she served as the founding chair of the portfolio management team, formed and executed business development strategy, drove global capital investment plans, and played a key role in capital market financings. In addition, she worked to drive the transformative growth of the company and support its evolution into a fully integrated business by developing and providing ongoing management of critical functions, including portfolio strategy, corporate development, commercial strategy, international, technical operations, corporate affairs, and information systems. Prior to joining Blueprint Medicines, Kate held leadership roles focused on building emerging, high-growth companies and advancing the development of innovative therapies in oncology and rare diseases as Vice President, Rare Diseases and Oncology Program Leadership at Idera Pharmaceuticals, Head of Commercial Development at Sarepta Therapeutics, Executive Director of Commercial Development at PTC Therapeutics, and roles in both corporate development and project management at Genzyme. She holds a B.A. from Wesleyan University with a double major in Biochemistry/Molecular Biology and Economics and an M.B.A. from Harvard Business School. Kate currently serves as chair of the board of directors at Fulcrum Therapeutics and is a member of the audit and compensation committees.

As hospitals and health care companies are racing to implement large language models like ChatGPT into their businesses, STAT reporter Casey Ross joins us to explain what experts want the world to know before embracing generative AI. We also discuss the latest twist for Sarepta Therapeutics and the quest to develop more potent weight loss medicines.

Layoffs have hit biopharma hard over the last year and a half. In this episode Fierce's Max Bayer and Gabrielle Mason will discuss the recent trends. We'll also cover a gene therapy by Sarepta Therapeutics, Abbott's FDA approval, plus this week's headlines.  To learn more about the topics in this episode:  Layoffs continue to batter biotech, with Big Pharmas piling on the pain Abbott's spinal cord stimulators score FDA approval to treat nonsurgical back pain Theranos' Elizabeth Holmes ordered to report to prison amid appeal, fined $452M in restitution alongside Sunny Balwani UPDATED: FTC sues to block Amgen's $28B Horizon buy in alarming sign for biopharma M&A UPDATED: FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy "The Top Line" is produced by senior podcast producer Teresa Carey. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.See omnystudio.com/listener for privacy information.

Jared Holz, biotech strategist at Mizuho Securities, joins us to explain why federal regulators are suing to block Amgen's $28 billion merger with Horizon Therapeutics and what it means for the drug industry. We also discuss the latest on Sarepta Therapeutics and its gene therapy for Duchenne muscular dystrophy, a turnaround story in the making at the Belgian drug maker Galapagos, and the effort to secure approval for a maternal RSV vaccine.

Das Buch zum Podcast. JETZT vorbestellen. SAP hat keine KI. Microsoft schon und bald auch Activision Blizzard - zumindest laut EU. Apropos haben: Sarepta Therapeutics könnte bald eine Zulassung für ein neues Medikament haben und die Börse freut's. Shopify für Landwirte in Afrika? Tingo Group (WKN: A2JQ1Y)! Vergesst Airbus und Boeing. Embraer (WKN: A1C2PZ) ist der Flugzeug-King. Zumindest regional & privat, aber leider sind keine Gewinne am Start. Diesen Podcast vom 16.05.2023, 3:00 Uhr stellt dir die Podstars GmbH (Noah Leidinger) zur Verfügung. Learn more about your ad choices. Visit megaphone.fm/adchoices

We discuss a Senate hearing that put major pharma CEOs in the same room with the middlemen they love to blame for rising drug prices, with mixed results. We also explain what is a massive week for Sarepta Therapeutics, the FDA, and the future of gene therapies for rare diseases, plus the latest news in the life sciences.

Our colleague Helen Branswell joins us to explain the latest news on Covid-19 boosters and the implications of a Marburg outbreak in Equatorial Guinea. Then we dive into the long history of a gene therapy from Sarepta Therapeutics, a polarizing medicine that promises to change the lives of patients with Duchenne muscular dystrophy. We also break down the rest of the week's biggest news in biopharma, including some billion-dollar deals and the retirement of perhaps the most storied executive in the business.

We discuss the theatrical merits and actual implications of Moderna CEO Stéphane Bancel's appearance before a committee led by Sen. Bernie Sanders. We also talk about the latest news in the life sciences, including Regeneron Pharmaceuticals' latest data for its powerhouse drug Dupixent, an about-face for Sarepta Therapeutics, and how the FDA appears to view biomarkers in neurological diseases.

Lisa Borland, Vice President of Global Medical Affairs at Sarepta Therapeutics, discusses the clinical development program evaluating the safety and efficacy of SRP-9001, an investigational gene transfer therapy for Duchenne muscular dystrophy. Data from this program supported the U.S. Food and Drug Administration's decision to accept and file a Biologics License Applications (BLA) for SRP-9001, which has a regulatory action date of May 29, 2023.Duchenne muscular dystrophy is the most common and most severe form of muscular dystrophy. It is caused by mutations in the DMD gene that lead to loss of dystrophin and progressive muscle loss. Symptoms of muscle loss most often appear between the ages of 3 to 5 years, and most children with this disease will be wheelchair dependent by their early teens.

In this episode, we are exploring limb-girdle muscular dystrophy (LGMD). Joining us for this conversation are two experts, Dr. Louise Rodino-Klapac, and genetic counselor Livija Medne.Dr. Louise Rodino-Klapac is the Executive Vice President, Head of R&D and Chief Scientific Officer at Sarepta Therapeutics who has 15 years of experience researching and studying LGMD. She is renowned for her work in molecular genetics and gene therapy. Her pioneering research is the foundation for five of our investigational limb-girdle muscular dystrophy (LGMD) programs. Hear from Dr. Rodino-Klapac about LGMD, the importance of knowing your subtype and the basics of gene therapy!Livija Medne is a Senior Genetic Counselor and Systems Director of Genetic Counseling at the Children's Hospital of Philadelphia. She has 15+ years of experience in pediatric neuromuscular diagnoses, including LGMD. She co-chairs and is one of the course directors of the Curriculum Committee at the University of Pennsylvania Genetic Counseling Program. In addition, Livija is an advocate for the professional development of junior genetic counselors, having co-founded the first Genetic Counselor Mentorship committee at CHOP.On This Episode We Discuss:Limb-girdle muscular dystrophy (LGMD)Which muscles are typically are affected first Symptoms that individuals with LGMD experience and when symptoms usually startThe prevalence of LGMD compared to Duchenne Muscular DystrophyHow people are diagnosed with LGMDWhy genetic testing is important to determine specific subtypes of LGMDGene therapy programs for the most common subtypesThe goal of the gene therapyWhen FDA-approval for LGMD gene therapy can be expected How patients can gain access to the gene therapy program and how health care providers can refer patientsBusting myths about LGMD If you'd like to take a deeper dive after listening to today's episode, follow these links to learn more about limb girdle muscular dystrophy, gene therapy, and genetic testing for LGMD. You can also visit raregenomes.org and limbgirdle.com to learn more! And be sure to follow Dr. Louise Rodino-Klapac on Twitter!You can also check out this installment of the PhenoTips Speaker Series to hear Kira interview Livija Medne about genetic counselors in leadership positions. Stay tuned for the next new episode of DNA Today on March 10th, 2023! New episodes are released every Friday. In the meantime, you can binge over 225 other episodes on Apple Podcasts, Spotify, streaming on the website, or any other podcast player by searching, “DNA Today”. Episodes since 2021 are also recorded with video which you can watch on our YouTube channel. DNA Today is hosted and produced by Kira Dineen. Our social media lead is Corinne Merlino. Our video lead is Amanda Andreoli. Our Outreach Intern is Sanya Tinaikar. Our Social Media Intern is Kajal Patel. And our Graphic Designer Ashlyn Enokian.See what else we are up to on Twitter, Instagram, Facebook, YouTube and our website, DNAToday.com. Questions/inquiries can be sent to info@DNAtoday.com. Which muscular dystrophy causes weakness of the muscles typically starting around the hips and shoulders? That would be limb girdle muscular dystrophy, or LGMD. LGMD is a group of neuromuscular diseases caused by mutations in genes responsible for proteins critical for muscle function, regulation, and repair1-3. Sarepta is a global biotechnology company working on engineering precision genetic medicine with the goal of changing the lives of people living with rare muscular dystrophies. Their multi-platform Precision Genetic Medicine Engine includes gene therapy, RNA and gene editing approaches. Oh that reminds me, ACMG is in March and Sarepta will be at booth 504. You can also head over to limbgirdle.com to learn more. (Sponsored) 1. Murphy AP and Straub V. J Neuromusc Dis. 2015;2(suppl. 2):S7-S19.2. Liewluck T and Milone M. Muscle Nerve. 2018;58(2):167-77.3. McNally EM. The Sarcoglycans. In: Landes Bioscience. 2000–2013.Surely you have heard of whole genome sequencing, but what about rapid and ultra-rapid whole genome sequencing? This is an emerging method of diagnosing genetic conditions for quick management. PerkinElmer Genomics offers this incredibly valuable test, which can be life saving for ill babies and kids. Learn more in our full episode (#226) with PerkinElmer Genomics. You can visit perkinelmergenomics.com for more information. (Sponsored)Surely you have heard of whole genome sequencing, but what about rapid and ultra-rapid whole genome sequencing? This is an emerging method of diagnosing genetic conditions for quick management. PerkinElmer Genomics offers this incredibly valuable test, which can be life saving for ill babies and kids. Learn more in our full episode with PerkinElmer Genomics on here, DNA Today! You can visit perkinelmergenomics.com for more information, the link is also available in the show notes and on our website DNAtoday.com. (Sponsored)I've enjoyed recording a few episodes about epigenetics, one of the interviews where I learned the most was with the Diagnostic Labs at the Greenwood Genetic Center. They taught me about EpiSign which is a novel clinically validated test that analyzes methylation. I just learned that since this episode in 2021, verison 4 of EpiSign has been released which has expanded to include over 70 conditions. If you are attending ACMG this month stop by booth 607 to chat with Greenwood Genetics. In the meantime brush up on your epigenetics by listening to Episode #145 of DNA Today and visit GreenwoodGeneticCenter. (Sponsored)

Educational Objectives Recall the pathophysiology and clinical manifestations of Duchenne muscular dystrophy (DMD), as well as the burden of DMD and its impact on patient quality of life List the latest updates for the safety and efficacy data of new and approved disease-modifying therapies in the treatment of DMD   Faculty: Amanda Hickman, PharmD, MPH, MSCS Central Support Pharmacist – Neuro/Psych/Sleep Trellis Rx Atlanta, Georgia   Moderator: Jessica L. Kerr, PharmD, CDCES Associate Dean – Office Professional and Student Affairs Professor/Clinical Pharmacist Southern Illinois University Edwardsville School of Pharmacy Edwardsville, Illinois   Amanda Hickman, PharmD, MPH, MSCS, and Jessica L. Kerr, PharmD, CDCES, have no financial relationships with commercial interests to disclose. Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 0.75 contact hours (0.075 CEU) under the ACPE universal activity number 0290-0000-22-613-H01-P. The activity is available for CE credit through December 22, 2023.   This activity is supported by an educational grant from Sarepta Therapeutics.

Educational Objectives Recall the pathophysiology and clinical manifestations of Duchenne muscular dystrophy (DMD), as well as the burden of DMD and its impact on patient quality of life List the latest updates for the safety and efficacy data of new and approved disease-modifying therapies in the treatment of DMD   Faculty: Amanda Hickman, PharmD, MPH, MSCS Central Support Pharmacist – Neuro/Psych/Sleep Trellis Rx Atlanta, Georgia   Moderator: Jessica L. Kerr, PharmD, CDCES Associate Dean – Office Professional and Student Affairs Professor/Clinical Pharmacist Southern Illinois University Edwardsville School of Pharmacy Edwardsville, Illinois   Amanda Hickman, PharmD, MPH, MSCS, and Jessica L. Kerr, PharmD, CDCES, have no financial relationships with commercial interests to disclose. Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 0.75 contact hours (0.075 CEU) under the ACPE universal activity number 0290-0000-22-613-H01-P. The activity is available for CE credit through December 22, 2023.   This activity is supported by an educational grant from Sarepta Therapeutics.

Zu Wochenbeginn knüpfte der Dow Jones an seine Verluste vom Freitag an. Gestern gab er 1,1 Prozent ab. Der Technologieindex Nasdaq 100 verlor hingegen nur 0,5 Prozent.

The FDA has given Sarepta Therapeutics the green light for its phase 2 Duchenne muscular dystrophy clinical trial after slapping the biotech with a clinical hold in June. The hold came after a grade 3 adverse event was reported in the midstage trial. Now, only a few months later, the agency has lifted the hold, and we'll discuss why there was a quick turnaround on the hold and what to expect next.  To learn more about topics in this episode:  Sarepta picks up momentum as FDA lifts hold on phase 2 DMD trial  As EU challenge intensifies, Illumina clocks a stateside win in FTC antitrust case over Grail buy  EU blocks Illumina's yearslong quest for Grail  Philips recalls 17M CPAP and BiPAP machine masks due to potential magnet interference  GSK helps SpringWorks double payday from boosted combo deal as it also reels $225M in new funding  BMS-backed Arsenal Bio pulls trigger on $220M series B, touting potential fleet of gene-edited cell therapies  Amylyx's ALS drug faces tough FDA scrutiny ahead of 2nd advisory panel meeting The Top Line is produced by senior multimedia producer Teresa Carey with managing editor Querida Anderson and senior editors Annalee Armstrong, Ben Adams, Conor Hale and Eric Sagonowsky. The sound engineer is Caleb Hodgson. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.See omnystudio.com/listener for privacy information.

Chris Garabedian is the Chairman & CEO of Xontogeny where he supports companies with cutting-edge technologies from early development through clinical proof of concept. Chris has decades of leadership experience in the biopharmaceutical industry. From 2011 to 2015, Chris led the turnaround of Sarepta Therapeutics as CEO. Prior to that, Chris served as VP of Corporate Strategy at Celgene and has held multiple executive roles at Gilead across corporate development, marketing, and medical affairs. Chris started his career in the biopharmaceutical industry as a consultant and holds a BS in Marketing from the University of Maryland.Thank you for listening!BIOS (@BIOS_Community) unites a community of Life Science innovators dedicated to driving patient impact. Alix Ventures (@AlixVentures) is a San Francisco based venture capital firm supporting early stage Life Science startups engineering biology to create radical advances in human health.Music: Danger Storm by Kevin MacLeod (link & license)

In this industry talk, Chris Garabedian, Chairman and CEO of Xontogeny LLC, discusses his career and gives advice to KGI students. Since 2009, Garabedian has been one of KGI's most engaged champions. He currently serves on the Executive Committee of the KGI Corporate Relations Board, has sponsored multiple TMPs, and has hired several KGI alumni. Garabedian has a robust track record of leadership in industry, including serving as CEO of Sarepta Therapeutics prior to founding biotech accelerator Xontogeny. As evidenced by his multiple board appointments, he is a sought-after expert and has a prolific record for contributing to companies and organizations to support the advancement of science and the development of the biotech industry.  

Is "flexible manufacturing" just another empty buzz concept concocted by the biopharma equipment marketing community, or does it demonstrate real benefit to bioprocess pros and their companies? On this episode, we sit down with Sarepta Therapeutics Director of Pharmaceutical Engineering Brian Winstead and Project Farma VP Tony Khoury to discuss what flexible manufacturing means, where it works, and where it doesn't. Listen in as Winstead shares the flexible manufacturing philosophy at Sarepta. 

Rozhovor s Janem Šumberou, analytikem z Odyssey 44 a také řečníkem České investiční konference 2021 o jeho investiční strategii a jak ji ovlivnilo dění kolem COVID-19, proč vsadil na biometan nebo zefektivnění procesů ve zdravotnictví, a co si myslí o akciích Avalara,  Tencent Music Entertainment Group, Iqvia a dalších.AKCIE: ULTA, XBC, GNLN, SBUX, NOW, IQV, FND, FIVE, AVLR, WING, TME, WIZZ, AF, SRPT, JUMP, HCAT, OPRXBIO: Jan Šumbera pracuje jako analytik ve family office Odyssey 44, kde se specializuje na veřejně obchodované společnosti západní Evropy a USA. Dříve pracoval jako akciový analytik pro společnosti Patria a Česká spořitelna. Vystudoval VŠE v Praze se zaměřením na Narodní hospodářství a finanční management. Úspěšně složil všechny zkoušky CFA programu.CO SE V PODCASTU DOZVÍTE:Family office Odyssey 44 a jejich projekty (2:46)Ulta Beauty (7:14) --> (Honzovu prezentaci z České investiční konference 2019 si můžete stáhnout zde)Inovativní prvky v segmentu (15:29)Xebec Adsorption a Greenlane Holdings a proč biometan (17:12)Propad portfolia a teorie chaosu (24:40)Starbucks (27:11)ServiceNow (30:38)Iqvia (34:40)Floor & Decor Holdings (41:19)S kým konzultuje své investiční nápady (42:35)Five Below (44:34)Avalara (48:20)Odprodej akcií Wingstop, Nike (50:52)Prázdninové uvažování  4 investiční témata, na která se zaměřoval (53:43)Firmy z efektňující procesy ve zdravotnictví a OptimizeRX Corporation, Health Catalyst (55:40)Sarepta Therapeutics, Leveljump Healthcare Corp. (58:15)Wizzair, Air France (1:00:00) --> (Shlédněte Honzovu přednášku společnosti Wizzair, kterou představil na České investiční konferenci 2020)Tencent Music Entertainment Group (1:03:19)Zhodnocení loňského roku (1:06:21)Největší investiční chyba (1:07:00)Investiční strategie (1:07:31)Trendy, které nyní sleduje (1:12:52)ZAJÍMAVÉ ODKAZY:Odkaz na Honzův investiční deník z roku 2020Peter C. Oppenheimer: The Long Good Buy: Analysing Cycles in MarketsJan-Benedict Steenkamp, Laurens Sloot: Retail Disruptors: The Spectacular Rise and Impact of the Hard DiscountersWilliam Green: Richer, Wiser, Happier: How the World's Greatest Investors Win in MarketsNenechte si ujít letošní ročník České investiční konference, který proběhne 8. a 9. listopadu 2021 online. Program naleznete zde. Partnerem podcastu je Strike.Market, alternativní data pro vaše investice.Vaše zpětná vazba je pro mě důležitá. Máte-li k podcastu jakékoliv poznámky, zašlete mi je, prosím, na lenka@schanova.com. Děkuji. 

In this episode of Women @ RopesTalk, hosted by IP transactions partner Megan Baca, life sciences partner Hannah Freeman interviews Cris Rothfuss, senior vice president of corporate transactions and deputy general counsel at Sarepta Therapeutics, a global biotechnology company focusing on rare diseases. An engaging storyteller, Cris talks about her unusual career trajectory, involving stops in private practice, academia and the biotech industry. She also shares her approach to mentoring—in her mind, a two-way street. And in thinking about leading teams, Cris reflects on a time in her life when her leadership skills were truly tested: when she orchestrated a 5,000-mile, three-month cross-country bike ride called The REAL Ride with four other riders to raise money for local schools.

In der heutigen Folge „Alles auf Aktien“ berichten die Finanzjournalisten Anja Ettel und Holger Zschäpitz über die Folgen der Landtagswahlen von Sachsen-Anhalt für die Börsen, über die Verlierer des historischen G7-Steuerdeals und erklären den Reddit-Hype um AMC. Außerdem geht es um Amazon, Apple, Facebook, Netflix, Münchener Rück, Infineon, Gamestop, Cineworld, iShares NASDAQ Biotech ETF (WKN: A2DWAW), Biogen, Eisai, Eli Lilly, Sarepta Therapeutics, Sage Therapeutics. "Alles auf Aktien" ist der tägliche Börsen-Shot aus der WELT-Wirtschaftsredaktion. Die Wirtschafts- und Finanzjournalisten Anja Ettel und Holger Zschäpitz diskutieren über die wichtigsten News an den Märkten und das Finanzthema des Tages. Außerdem gibt es jeden Tag eine Inspiration, die das Leben leichter machen soll. In nur zehn Minuten geht es um alles, was man aktuell über Aktien, ETFs, Fonds und erfolgreiche Geldanlage wissen sollte. Für erfahrene Anleger und Neueinsteiger. Montag bis Freitag, ab 6 Uhr morgens. Wir freuen uns an Feedback über aaa@welt.de. Disclaimer: Die im Podcast besprochenen Aktien und Fonds stellen keine spezifischen Kauf- oder Anlage-Empfehlungen dar. Die Moderatoren und der Verlag haften nicht für etwaige Verluste, die aufgrund der Umsetzung der Gedanken oder Ideen entstehen. Für alle, die noch mehr wissen wollen: Holger Zschäpitz können Sie jede Woche im Finanz- und Wirtschaftspodcast "Deffner&Zschäpitz" hören. +++Werbung+++ Hier geht's zur App: Scalable Capital ist der Broker mit Flatrate. Unbegrenzt Aktien traden und alle ETFs kostenlos besparen – für nur 2,99 € im Monat, ohne weitere Kosten. Und jetzt ab aufs Parkett, die Scalable App downloaden und loslegen. Hier geht's zur App: https://bit.ly/3abrHQm

FirstWord Pharma PLUS editors Simon King and Becky Simon discuss Pfizer and Moderna's COVID-19 vaccine revenue forecasts for the year ahead, controversy around the potential lifting of intellectual property protection on COVID-19 vaccines and new, early-stage data for Sarepta Therapeutics' latest Duchene muscular dystrophy therapy.

Leaders in healthcare are facing a world that is anything but the status quo. In today’s episode, Katie Lawrence, MHA, CMPE, Director, Ambulatory Optimization & Integration, Prisma Health, examines how leaders can address change in their organizations to become more successful in engaging team members, physicians, and other stakeholders. As Katie says, leadership begins with self, and she talks about how leaders can identify their reactions to changes and how to authentically accept those reactions while understanding that others may not view the same change in the same way. Katie she says one great source she often turns to is Dr. Brené Brown, a research professor who has spent the past two decades studying courage, vulnerability, shame, and empathy. You can learn more about her teachings at https://brenebrown.com. Thanks to Sarepta Therapeutics and to Phreesia for sponsoring this week’s show. Read a new white paper from Sarepta Therapeutics at mgma.com/sarepta. And to find out how Phreesia’s contactless intake workflows are helping practices eliminate the waiting room, visit https://bit.ly/3lm05MV. ​If you like the show, please rate and review it wherever you get your podcasts. If you have topics you'd like us to cover or experts you'd like us to interview, email us at podcasts@mgma.com, or reach out to MGMA Sr. Editor and MGMA Insights podcast host Daniel Williams on Twitter at twitter.com/MGMADaniel. MGMA Insights is presented by Decklan McGee, Rob Ketcham and Daniel Williams.

Leadership in times of crisis is very different than during normal day-to-day operations. Today, we’re joined by Aimee Greeter, Senior Vice President with the Coker Group, who talks about key strategies to managing complex and stressful situations, and how best to pick up the pieces in a way that guarantees future stability and success moving forward. Thanks to Sarepta Therapeutics and to R1RCM for sponsoring this week’s show. Read a new white paper from Sarepta Therapeutics at mgma.com/sarepta. Access a thought leadership discussion with R1 and three physician group leaders to hear key learnings on sustaining positive practice performance. To attend this free educational program, please visit R1RCM.com/MGMA. ​If you like the show, please rate and review it wherever you get your podcasts. If you have topics you'd like us to cover or experts you'd like us to interview, email us at podcasts@mgma.com, or reach out to MGMA Sr. Editor and MGMA Insights podcast host Daniel Williams on Twitter at twitter.com/MGMADaniel. MGMA Insights is presented by Decklan McGee, Rob Ketcham and Daniel Williams.

In this episode, MGMA Data Analyst II Ryan Lawler explains the criteria used in the Better Performer process, and Steve Sinclair, CFO of Graves-Gilbert Clinic, discusses how data analysis has taken his practice to a new level and offers steps other practices can take to become a Better Performer. Thanks to Sarepta Therapeutics and to R1RCM for sponsoring this week’s show. Read a new white paper from Sarepta Therapeutics at mgma.com/sarepta. Access a thought leadership discussion with R1 and three physician group leaders to hear key learnings on sustaining positive practice performance. To attend this free educational program, please visit R1RCM.com/MGMA. ​If you like the show, please rate and review it wherever you get your podcasts. If you have topics you'd like us to cover or experts you'd like us to interview, email us at podcasts@mgma.com, or reach out to MGMA Sr. Editor and MGMA Insights podcast host Daniel Williams on Twitter at twitter.com/MGMADaniel. MGMA Insights is presented by Decklan McGee, Rob Ketcham and Daniel Williams.

  In this episode, Mark talks with Chris Garabedian, Chairman and CEO of Xontogeny, which he founded in 2016 to support multiple promising technologies from early development through clinical proof of concept. In 2017, Chris joined Perceptive Advisors to develop their Venture Fund strategy and launched the Perceptive Xontogeny Venture Fund in 2018 to support early stage companies seeded and incubated at Xontogeny and other ventures. Chris has a broad base of experience and a track record of success over his decades long experience in the biopharma industry.  He served as the President and CEO of Sarepta Therapeutics from 2011 to 2015, overseeing the turnaround of a company that is now a commercial stage leader in the genetic technology space after leading the development of the company’s Duchenne Muscular Dystrophy program. Prior to Sarepta, Chris led Corporate Strategy for Celgene from 2007 to 2010. Prior to Celgene, Chris served in a number of global commercial and corporate development leadership roles at Gilead from 1997 to 2005. Chris serves on a number of Boards of life sciences companies and speaks at industry conferences on a wide range of important issues.  Chris is also on the Board of Directors of MassBio and serves as a Senior Advisor for the Boston Consulting Group. Chris earned his B.S. in Marketing from the University of Maryland. Mark and Chris discussed a range of topics, including: What led Chris to found Xontongeny - what was missing in the marketplace that Xontongeny delivers What Chris finds effective founders have in common that increases the likelihood they’ll succeed?  The kinds of red flags that stand out that may lead Chris and his team to decide to take a pass on investing in a startup How Chris thinks the pandemic will shape program development moving forward The skill sets or expertise founders should have beforethey walk in the door to speak to an accelerator or other potential funders  Some of the key services Xontongeny offers its scientific founders that are particularly important to helping them move successfully through the program development lifecycle, hopefully to approval For scientists and entrepreneurs interested in translating their science to a viable commercial product, how should they begin to prepare and what should their mindset be as they think about working with a company like Xontongeny

Patients with rare diseases may soon have better access to treatments. A second installment to the 21st Century Cures Act is in the works that would make improvements to telemedicine and digital health regulations. Find out what this means for the rare disease community in this interview with Diane Berry, Ph.D. And hear Kate’s interview with Lan Sena, a healthcare advocate who has been battling Hodgkin’s lymphoma for the past ten years. Guest:Diane Berry, Ph.D.Senior VP, Global Health Policy, Government and Patient Affairs, Sarepta TherapeuticsDiane joined Sarepta Therapeutics in December of 2011 and serves as Vice President, Global Policy, Government & Patient Affairs. She engages policymakers at the federal, state, and local levels, as well as patient advocacy organizations. She works to advance critical policies related to newborn screening, regulatory policy, and reimbursement and access, with the goal of expediting development and patient access to genetic-based therapies for rare diseases.Previously, Dr. Berry served in leadership roles for the federal government in both the legislative and executive branches, overseeing and implementing science, technology and public health activities. She served as a Subcommittee Staff Director and Senior Professional Staff Member for the U.S. House of Representatives Committee on Homeland Security and as Chief Scientist and Senior Biodefense Advisor at the Department of Homeland Security inside its Office of Health Affairs. Dr. Berry was also a Senior Science Advisor at McKenna, Long, and Aldridge, and a Science and Technology Policy Advisor and Fellow within the Department of Defense through the American Association for the Advancement of Science.Dr. Berry earned her Ph.D. in chemical engineering from Northwestern University and her B.S. and M.S. in chemical/biochemical engineering from Tufts University. Hosts:Terry Wilcox, Executive Director, Patients RisingDr. Robert Goldberg, “Dr. Bob”, Co-Founder and Vice President of the Center for Medicine in the Public Interest.Kate Pecora, Field Correspondent Links:Diane Berry, Ph.D. Be The MatchDoctors can still prescribe HCQ to patients, says U.S. Health Secretary Patients Rising Concierge Need help?The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands. Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.orgHave a question or comment about the show, want to suggest a show topic or share your story as a patient correspondent?Drop us a line: podcast@patientsrising.orgThe views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising.

What will become of biotech in 2019? What's the legacy of Sarepta Therapeutics? And how do you say "overhyped" in Mandarin?

Gary Charobonneau joins us today as the SVP of Regulatory Affairs at Sarepta Therapeutics. Sarepta, based in Cambridge, MA is focused on the discovery of unique rNA and gene therapy targeted therapeutics for a variety of neuromuscular rare diseases. Gary has over 25 years of experience within Regulatory Affairs. He was instrumental in building out the Regulatory team at Allergan and has held Executive roles with many well-known organizations (e.g. Amgen, Roche BioScience, Novo Nordisk, etc). Listen in as Gary outlines how he got to the Executive role he holds today and his advice for those aspiring to do the same.

Big news sent Foundation Medicine and Sarepta Therapeutics soaring this week. Meanwhile, PTC Therapeutics investors endured a roller coaster ride. Stocks: FMI, SRPT, PTCT 

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Duchenne Muscular Dystrophy, a rare genetic disease, causes progressive muscle wasting that slowly robs people of abilities and leads to death. In recent years, much attention has focused on the use of antisense oligonucleotides to bypass defective portions of the exon that codes for the dystrophin gene to restore its production. Dystrophin is a protein that is essential to healthy muscle. Summit Therapeutics is taking a different approach. Instead of restoring dystrophin, Summit is developing a drug that activate utrophin, a related protein that serves a similar function to dystrophin during fetal development, but then shuts off. We spoke to Glyn Edwards, CEO of Summit, about the company’s utrophin activator ezutromid, its licensing deal with Sarepta Therapeutics, and why, unlike the exon-skipping drugs that target specific subpopulaitons of Duchenne patients, ezutromid could provide benefits to patients broadly.

1) Glucocorticoid-associated worsening in reversible cerebral vasoconstriction syndrome2) What's Trending: Upcoming changes regarding the Neurology® Resident & Fellow section 3) Topic of the Month: Neuromuscular topicsThis podcast begins and closes with Dr. Robert Gross, Editor-in-Chief, briefly discussing highlighted articles from the January 17, 2017 issue of Neurology. In the first segment, Dr. Andy Southerland talks with Dr. Aneesh Singhal about his paper on glucocorticoid-associated worsening of reversible cerebral vasoconstriction syndrome. Dr. Ted Burns talks with Dr. Roy Strowd about upcoming changes regarding the Neurology Resident & Fellow section for our “What's Trending” feature of the week. In the next part of the podcast, Dr. Ted Burns focuses his interview with Dr. Volker Straub on limb-girdle dystrophy. Disclosures can be found at Neurology.org.DISCLOSURES: Dr. Southerland serves as Podcast Deputy Editor for Neurology; receives research support from the American Heart Association-American Stroke Association National Clinical Research Program, American Academy of Neurology, American Board of Psychiatry and Neurology, Health Resources Services Administration and the NIH; has a provisional patent application titled: “Method, system and computer readable medium for improving treatment times for rapid evaluation of acute stroke via mobile telemedicine;” and gave legal expert review. Dr. Aneesh Singhal has served on the scientific advisory boards of Biogen and DSMB; has served on the editorial board of Medical Gas Research; has received publishing royalties for the book Reversible Cerebral Vasoconstriction Syndromes; has been a consultant to Biogen; has acted as an event adjudicator for the Thrombolysis in Myocardial Infarction (TIMI) Trial Group; has received research support from Boehringer Ingelheim, NIH-NINDS, the American Academy of Neurology, UpToDate, and Medlink; and has served as a medicolegal expert witness for individual cases concerning stroke. Dr. Burns serves as Podcast Editor for Neurology®; and has received research support for consulting activities with UCB, CSL Behring, Walgreens and Alexion Pharmaceuticals, Inc. Dr. Strowd serves on the editorial team for the Neurology® Resident and Fellow Section. Dr. Straub has served on the scientific advisory boards for Pfizer, Italfarmaco, Audentes Therapeutics, Bristol-Myer Squibb, Summit Therapeutics, Tivorsan, and the Nationwide Children's Hospital; has received travel funding and speaker honoraria from Sanofi Genzyme; has served on the editorial boards of Neuromuscular Disorders, the Journal of Neuromuscular Diseases, and PLoS Currents Muscular Dystrophy; has been a consultant for Sanofi Genzyme; and has received research support from Sanofi Genzyme, BioMarin, Ionis Pharmaceuticals, Sarepta Therapeutics, Ultragenyx, the European Commission, the UK Medical Research Council, Newcastle University, the Parent Project Muscular Dystrophy, the Association Fracaise Contre les Myopathies, the LGMD2I Research Fund, the Wellcome Trust, the Sylvia Aitken Charitable Trust, Muscular Dystrophy UK, and Action Medical Research.All other participants have no disclosures.

The controversial approval of Sarepta Therapeutics eteplirsen to treat a certain form of Duchenne muscular dystrophy has been viewed as a major victory for patient advocates. Advocates aggressively lobbied the U.S. Food and Drug Administration to grant approval for the drug despite a weak data package presented by the company. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research overrode staff to greenlight the drug. We spoke to Debra Miller, president, CEO, and co-founder of CureDuchenne, about the significance of the approval, her organization’s venture philanthropy model, and what other hope the Duchenne drug pipeline may hold for patients.

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While much of the attention of the Duchenne Muscular Dystrophy community has been focused on the recent FDA advisory committee review of Sarepta Therapeutics’ experimental drug Eteplirsen, Marathon Pharmaceuticals has been working to advance its own DMD drug candidate to the FDA. The company expects this month to file an application with the agency for approval to market Deflazacort, a corticoid steroid. We spoke to Tim Cunniff, head of research and development for Marathon Pharmaceuticals, about why the company thinks Deflazacort represents an improvement over existing therapies, what the company has learned from Sarepta’s experience, and its new effort to develop a precision medicine program in Duchenne.

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