Gene-Based Therapy for SMA: Translating Research to Clinical Practice

Sunday, March 8, 2020

9:15 AM – 9:45 AM

Location: Gatlin E 3/4

Speaker(s)

Susan Apkon, MD

Visiting Professor and Vice Chair
University of Colorado School of Medicine

Disclosure: Disclosure information not submitted.
W. David Arnold, MD

Associate Professor
The Ohio State University

Disclosure: Disclosure information not submitted.
Stacy Suskauer, MD

Associate Professor
Kennedy Krieger Institute/ Johns Hopkins School of Medicine

Disclosure: Disclosure information not submitted.

Exciting progress over the last few years has resulted in the clinical approval of new gene-based therapies for Spinal Muscular Atrophy (SMA) including nusinersen and a viral vector-based treatment. Hear experiences initiating and maintaining programs to provide gene-based therapies to patients with SMA, as well as barriers/challenges faced.