Gene Treatment Panel and Q&A | Spinal Muscular Atrophy 2023 скачать в хорошем качестве

Gene Treatment Panel and Q&A | Spinal Muscular Atrophy 2023

This session covers gene therapy treatments for spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Key topics covered include managing thrombotic microangiopathy (TMA) side effects, approaches for unreliable families, use of steroids, measuring treatment efficacy especially in younger patients, and considerations around newborn screening and early treatment. The panelists share insights from their clinical experiences and raise open questions needing further research. Action items center on gathering more evidence around combination therapies, steroid regimens, assessing very young patients, and building infrastructure for newborn screening. View accreditation information and claim CME at https://stanford.cloud-cme.com/SMAOnl...