#CGIC2O24 Day 2, Session 2 was moderated by C-Path's Vice President of Rare and Orphan Disease programs, Collin Hovinga. The panel discussed: "A Potential New Framework to Accelerate Drug Development for Rare Diseases." The session emphasized the importance of data sharing in rare disease research, highlighting key initiatives and collaboration for drug development. Watch now: https://lnkd.in/gWfCksSu #CPath #RareDisease #DataSharing #DrugDevelopment
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#CGIC2O24 Day 2, Session 2 was moderated by C-Path's Vice President of Rare and Orphan Disease programs, Collin Hovinga. The panel discussed: "A Potential New Framework to Accelerate Drug Development for Rare Diseases." The session emphasized the importance of data sharing in rare disease research, highlighting key initiatives and collaboration for drug development. Watch now: https://lnkd.in/gAvY8A-g #CPath #RareDisease #DataSharing #DrugDevelopment
CGIC2024 Day 2: A Potential New Framework to Advancing Drug Development
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Business Development Manager, Gene Therapy CDMO Services - East @ Charles River Labs
With a staggering 7,000 rare diseases spanning various therapeutic areas, take a moment to discover the latest collaborations, frameworks, and portfolios designed to support drug development. Explore the extraordinary: https://okt.to/1e6Cl2 #cellandgenetherapies #CGT #advancedtherapies #raredisease #genetherapy
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With a staggering 7,000 rare diseases spanning various therapeutic areas, take a moment to discover the latest collaborations, frameworks, and portfolios designed to support drug development. Explore the extraordinary: https://okt.to/VXlq1J #cellandgenetherapies #CGT #advancedtherapies #raredisease
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With a staggering 7,000 rare diseases spanning various therapeutic areas, take a moment to discover the latest collaborations, frameworks, and portfolios designed to support drug development. Explore the extraordinary: https://okt.to/wuSb1U #cellandgenetherapies #CGT #advancedtherapies #raredisease
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With a staggering 7,000 rare diseases spanning various therapeutic areas, take a moment to discover the latest collaborations, frameworks, and portfolios designed to support drug development. Explore the extraordinary: https://okt.to/otKifp #cellandgenetherapies #CGT #advancedtherapies #raredisease
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Groundbreaking Workshop to Propel Rare Diseases Treatment Innovations: Reagan-Udall Foundation and Partners Reagan-Udall Foundation is organizing a workshop to discuss the utilization of natural history study and registry data in developing treatments for rare diseases. The workshop aims to bring together stakeholders like patient advocates, researchers, and industry representatives to address challenges and opportunities in this field. The participation of regulatory agencies highlights the importance of collaboration in advancing rare disease research. Natural history studies and patient registries are crucial tools for understanding and developing treatments for rare diseases. They provide insights into disease progression and collect standardized clinical data. Leveraging these tools can enhance drug development efforts and improve patient outcomes. For more details please click the link! https://lnkd.in/dsmKTaK9 #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical
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With a staggering 7,000 rare diseases spanning various therapeutic areas, take a moment to discover the latest collaborations, frameworks, and portfolios designed to support drug development. Explore the extraordinary: https://okt.to/jU3zZs #cellandgenetherapies #CGT #advancedtherapies #raredisease
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Business Development Manager Central/South Spain Charles River Laboratories
With a staggering 7,000 rare diseases spanning various therapeutic areas, take a moment to discover the latest collaborations, frameworks, and portfolios designed to support drug development. Explore the extraordinary: https://okt.to/0akMqB #cellandgenetherapies #CGT #advancedtherapies #raredisease
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𝐁𝐢𝐨𝐡𝐚𝐯𝐞𝐧'𝐬 𝐓𝐫𝐨𝐫𝐢𝐥𝐮𝐳𝐨𝐥𝐞 𝐒𝐡𝐨𝐰𝐬 𝐏𝐫𝐨𝐦𝐢𝐬𝐞 𝐢𝐧 𝐒𝐥𝐨𝐰𝐢𝐧𝐠 𝐒𝐂𝐀 𝐏𝐫𝐨𝐠𝐫𝐞𝐬𝐬𝐢𝐨𝐧 Biohaven Ltd. (NYSE: BHVN) Biohaven has announced positive topline results from Study BHV4157-206-RWE, demonstrating the efficacy of troriluzole in slowing disease progression in spinocerebellar ataxia (SCA) patients. The study achieved its primary endpoint, showing a 50-70% slower rate of decline compared to untreated patients, translating to a significant delay in disease progression over three years. Dr. Susan Perlman stated, "Troriluzole is the first treatment to show a delay in SCA progression, offering patients additional years of independence." The data also supports a New Drug Application submission to the FDA in Q4 2024. Biohaven remains committed to advancing treatments for rare diseases like SCA, aiming for commercialization in the U.S. by 2025, pending approval. #Biohaven #Troriluzole #SpinocerebellarAtaxia #SCA #FDAApproval #Neurodegeneration #RareDiseases #ClinicalTrials #Innovation
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Check out the article by Josh Thayer, General Counsel of the Jain Foundation, in the latest edition of LGMD News magazine published by the Speak Foundation. In Vol 4, Issue 2, Josh describes the roles of Surrogate Endpoints and Accelerated Approval in the drug approval process, which are very important for rare disease drug development. This is an insightful article offering an approachable explanation for the non-expert interested in learning about the drug approval process for rare diseases and how it differs from traditional drug approval. To read more on this topic please visit https://lnkd.in/dwW3T2Xe. Pages 28-31 are drafted by Josh Thayer.
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