Supported by angel donors and industry partners, our Therapeutic Accelerator Project promises to overcome obstacles to FDA approval and ensure that effective therapies get to our families faster.
Since the genetic disease mechanism was discovered in 2010, facioscapulohumeral muscular dystrophy (FSHD) research has picked up speed and caught momentum. Brilliant researchers have developed 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 Project.
Therapeutic Accelerator Project areas
- Global patient registry & natural history studies
- Biochemical markers and tools to measure therapy effect
- Trial endpoints and clinical outcomes accepted by FDA (and European EMA)
All of these areas require a coordinated effort to ensure that clinical trial measurement tools meet industry and FDA standards. All drug development companies will have an accepted way to measure the effectiveness of their drugs, and the most promising will go into clinical trials faster.
Interested in investing in this exciting project as an angel donor or industry partner? Let’s continue the conversation.
Contact: Mark Stone President and CEO
450 Bedford Street
Lexington, MA 02420