Therapeutic Accelerator

Brilliant researchers are developing therapies that improve—and sometimes reverse—the condition in a test tube, but there is a lack of accepted criteria to prove that a drug is effective in patients and deserving of FDA (and European EMA) approval. This is beginning to slow the drug development process down. Overcoming the hurdles is the only way to shorten the time it will take to get these therapies to our families. With every affected patient in mind, we have risen to this challenge and started our Therapeutic Accelerator Initiative.

In March 2019, the FSHD Society convened a landmark meeting attended by FDA regulators as well as researchers from seven biopharmaceutical companies, academic scientists who are leading the effort to prepare the field for clinical trials, and representatives of patient advocacy organizations including the MDA, Friends of FSH, and Chris Carrino Foundation. The workshop introduced the FDA to FSH muscular dystrophy–the impact of the disease on individuals, why scientists think FSHD is treatable, and the progress that has been made toward clinical trials–and sought input from the FDA on how to develop methods to measure treatment effects to meet the standards for FDA approval. The findings from the workshop are summarized in a white paper, Industry Collaborative Workshop for Therapy Development in FSHD.