Roadblocks Faced by Gene Therapies for Muscular Dystrophies
March 14th 2025John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.
Gene and Cell Therapy Approaches Hold Promise for Usher Syndrome
September 21st 2024In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.
Evaluating Spinal Muscular Atrophy Gene Therapy Zolgensma in Heavier and Older Patients
March 16th 2024Sandra P. Reyna, MD, the chief scientific advisor and head of global medical engagement for SMA at Novartis, discussed data from the phase 3b SMART clinical trial (NCT04851873) that were presented at MDA's 2024 conference.