The chief research officer of the Muscular Dystrophy Association discussed research needs that remain in the field in light of recent progress and approvals.
“I think we're going to have to develop some new strategies for people who progress past the therapeutic window. There are also plenty of other diseases, muscle diseases and other types of neuromuscular disease, for which therapies aren't as advanced. Sometimes, because it's a particularly tricky thing to tackle, like a dominant mutation in which gain of function can be a little tricky... Other times diseases are too rare for there to be a good commercial market.”
2023 has seen lots of progress in the treatment landscape of Duchenne muscular dystrophy, including the summer approval of delandistrogene moxeparvovec (marketed as Elevidys by Sarepta), the field’s first gene therapy. Progress notwithstanding, September was Duchenne Action Month and CGTLive is highlighting that more work always remains to be done. Future research to investigate may include combination therapies with stem cell therapy or other therapeutics.
CGTLive spoke with Sharon Hesterlee, PhD, chief research officer, Muscular Dystrophy Association (MDA) to learn more about the research needs that remain in the muscular dystrophy field despite unprecedented progress. This includes finding solutions for populations unable to benefit from gene therapy’s mechanism of preventing further degeneration as well as furthering research efforts in rarer muscular dystrophies or diseases with harder-to-target etiologies. Hesterlee also touched on the upcoming March 2024 MDA meeting and encouraged clinicians in the field to attend, and shared recent news on $8.6 million in grants the MDA recently received.