PCE

What’s New in the Treatment of Spinal Muscular Atrophy

Episode Summary

Dr. Julie Parsons and Dr. Vanessa Battista discuss current and emerging medication therapies for the treatment of spinal muscular atrophy (SMA).

Episode Notes

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, MD
Co-Director, Neuromuscular Clinic
Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
Children’s Hospital Colorado
Denver, Colorado

Vanessa Battista, DNP, MBA, RN, MS, CPNP-PC, CHPPN, FPCN
Senior Director of Nursing, Palliative Care
Psychosocial Oncology and Palliative Care
Dana-Farber Cancer Institute
Boston, Massachusetts

Supported by an educational grant from Biogen

Link to full program:
bit.ly/41kw2dK

Link to CME: Claim credit -
bit.ly/40fkDKD