View Now | Addressing the Gaps in Clinical Trial Readiness for FSHD
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.
Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 55% funded by the FDA/HHS, totaling $17,612,250, and 45% funded by non-government source(s), totaling $14,203,111. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.