Carrie Miceli, PhD, on the Potential of Needle Biopsy Findings to Improve DMD Gene Therapy
March 9th 2024The professor of microbiology, immunology, and molecular genetics at UCLA discussed how a better understanding of treatment impact on a cellular level could help improve future gene therapy approaches.
Paul Melmeyer, MPP, on Advocacy and Progress in Rare Neuromuscular Diseases From a Policy Standpoint
February 29th 2024In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.
Kevin Flanigan, MD, on the Impact of Neuromuscular Disease Gene Therapy Approvals
April 9th 2023The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.