Addressing the gaps in clinical trial readiness for FSHD

Описание: Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common myopathies affecting anywhere between 1:8,000 to 1:20,000 individuals. There are currently no effective therapies for this indication. The FSHD Society is a research-focused patient advocacy nonprofit organization dedicated to finding a treatment for this devastating disease while also empowering the patient community. With increasing interest by biopharmaceuticals in this indication, the FSHD Society is leveraging existing clinical data to help chart the natural history of the disease, promote the identification of suitable clinical endpoints and enhance patient stratification for upcoming trials. In this webinar, we will discuss the perceived gaps in clinical trial readiness and the various efforts the FSHD Society is taking to help accelerate clinical development.

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Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

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