Updates in clinical data for FDA-approved disease-modifying therapies for spinal muscular atrophy

Abstract

Spinal muscular atrophy (SMA) is a rare, diverse group of inherited neuromuscular disorders that cause degradation of the lower motor neurons, progressive muscle atrophy, and weakness. The natural history of SMA has changed significantly with an increased understanding of SMA pathophysiology and new technologies. As a result, affected individuals now have 3 disease-modifying therapies available for treatment. Evidence suggests that these novel agents are more effective when started early in the disease process. This reinforces the importance of newborn screening as a mechanism for early diagnosis. Pharmacists are highly valued members of the healthcare team who play a pivotal role in the SMA care team. Therefore, pharmacists must be up-to-date on SMA's medical management, including the most current efficacy and safety data to assist providers, caregivers, and patients in selecting these agents and ensuring patients with SMA receive optimal and timely medical care.