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.
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.