New MDA Program for Gene Therapy Development in Ultra-Rare Diseases: Sharon Hesterlee, PhD
At the 2023 MDA Conference, the executive vice president and chief research officer of MDA talked about a new program from the MDA to tackle ultra-rare neuromuscular diseases. [WATCH TIME: 5 minutes]
WATCH TIME: 5 minutes
“There are short term solutions and long-term solutions, we're probably ultimately going to need a long-term fix. That's going to involve changes in how the regulatory agencies review these ultra-rare diseases.”
Gene therapy has shown to be a feasible, but tricky, strategy to treat the underlying genetic cause of several
Recently, a session on the new program was presented at the
After the session, Hesterlee, the executive vice president and chief research officer of MDA, sat down with NeurologyLive® in an interview at the conference to discuss more on the highlights of her presentation. She talked about the role industry plays in the commercialization of drugs and the cost of therapies for neuromuscular diseases compared with rare diseases. In addition, she also summarized a potential treatment for patients with congenital myasthenic syndrome who experience episodic apnea.
REFERENCES
1. Muscular Dystrophy Association. MDA Kickstart Program. Accessed March 23, 2023. https://www.mda.org/research/kickstart
2. Hesterlee S. MDA Kickstart Program for Ultra-Rare Gene Therapy Development. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Ultra Rare Track session.
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