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.
John Ligon, MD, on Establishing a Roadmap for CAR-T Fertility Research
March 14th 2024The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed the importance of involving all relevant providers and patients in research to ensure the right questions are being asked.
Open-Label Studies Are Insufficient to Establish the Potential of CAR-T in Autoimmune Disease
March 13th 2024Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the need for randomized-control trials for CAR-T in lupus and other autoimmune diseases.
John Ligon, MD, on Fertility Outcomes in Patients Who Have Received CAR-T
March 11th 2024The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the need to set standard guidelines regarding potential fertility issues for CAR-T treatment.
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.