PJ Brooks, PhD, on Strategies to Facilitate NMD Therapy Development


The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

“One of the things that came up in the discussion that we've seen in other countries, is if there are other ways to incorporate research and clinical care. It's difficult, I know, in the United States, the way our healthcare system is set up, to do that as much as other countries, but I feel like, to the extent possible, there's an opportunity there. If it can be done in a rational way, without taking away the already valuable time of clinicians. That's something that we discussed as well.”

Athough neuromuscular diseases and muscular dystrophies saw a big win in 2023 with the approval of Elevidys (delandistrogene moxeparvovec; Sarepta Therapeutics), the first gene therapy for treating Duchenne muscular dystrophy (DMD) and a muscular dystrophy in general, thousands of neuromuscular diseases still lack disease-modifying therapies.

One focus of the recent 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, was discussing how to facilitate drug development and more therapies to underserved populations of neuromuscular disease

PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences, National Institutes of Health (NIH), participated in 1 such panel discussion during the conference. CGTLive® spoke with Brooks to learn more about some of the highlights of the session and topics that were discussed, including natural history studies, outcome measures, redosing with AAV, accelerated approval, and integrating research into clinical care.

Click here to view more coverage of the 2024 MDA Conference.

Hesterlee S, Brooks PJ, Byrne B, Moy J, McNally E. Panel Discussion on NMD in the Age of Therapy. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL.
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