Spinal Muscular Atrophy (SMA)


MDA 2022 Conference to Focus on Promising Therapies for SMA, DMD

Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) will take center stage early next year at the first in-person annual gathering of the Muscular Dystrophy Association (MDA) since March 2019. The 2022 MDA Clinical & Scientific Conference, set for March 13-16 in Nashville, Tennessee, is expected to attract between 700 and 800 people in…
treating spinal muscular atrophy

Treating Spinal Muscular Atrophy: Recent Clinical Insights

Several recently published studies may help enhance our treatment of spinal muscular atrophy (SMA). Review key points of some of them below. Evaluating a Novel Myostatin Inhibitor as an SMA Treatment A new article in Advances in Therapy describes a phase 1 trial of a monoclonal antibody intervention being developed as a treatment for SMA1.…

Patient Perspectives

SMA Clinical Insights

Rare Care Podcast

An interview with SMA researcher Adrian Krainer, PhD, from the Cold Spring Harbor Laboratory.