In this episode, Dr. Parsons and Dr. Battista discuss current and emerging medical therapies for the treatment of spinal muscular atrophy (SMA). They review safety, efficacy, and monitoring parameters of current agents, as well as adjuct therapies in the medication pipeline. This high-level overview includes:
- Review of FDA-approved medical therapies: nusinersen, onasemnogene abeparvovec-xioi, and risdiplam
- Discussion of the safety, efficacy, and monitoring parameters for FDA approved medications, as well as adjuctive agents for medication therapy complications
- Review of ongoing clinical trials on anti-myostatin medications and discussion of the role these agents would play if approved by the FDA
Presenters:
Julie Parsons, MDCo-Director, Neuromuscular ClinicHaberfeld Family Endowed Chair in Pediatric Neuromuscular DisordersProfessor of Clinical Pediatrics and NeurologyChildren’s Hospital ColoradoDenver, Colorado
Vanessa Battista, DNP, MBA, RN, MS, CPNP-PC, CHPPN, FPCNSenior Director of Nursing, Palliative CarePsychosocial Oncology and Palliative CareDana-Farber Cancer InstituteBoston, Massachusetts
Supported by an educational grant from Biogen
Link to full program:bit.ly/41kw2dK
Link to CME: Claim credit -bit.ly/40fkDKD
