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 neuromuscular diseases. To lower the barriers for ultra-rare gene therapies, the Muscular Dystrophy Association (MDA) created the Kickstart Program to support gene therapy programs. The program will put together a team to collaboratively assist selected projects into position for follow-on grant funding.1

Recently, a session on the new program was presented at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, chaired by Sharon Hesterlee, PhD. In addition to an overview of the program and its potential, the session also focused on opportunities to de-risk gene therapies, the current barriers to furthering the development these agents, collaborative framework and data management.2

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.

Click here for more coverage of MDA 2023.

1. Muscular Dystrophy Association. MDA Kickstart Program. Accessed March 23, 2023.
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.
Related Videos
Shahid Nimjee, MD, PhD
Peter J. McAllister, MD, FAAN
Video 6 - "Utilization of Neuroimaging in Alzheimer’s Disease"
Video 5 - "Contribution of Multiple Pathways to the Development of Alzheimer’s Disease"
Michael Levy, MD, PhD
Michael Levy, MD, PhD
Michael Kaplitt, MD, PhD
© 2024 MJH Life Sciences

All rights reserved.