Making a Difference in Patients With Spinal Muscular Atrophy: Improving Diagnosis and Treatment

Target Audience
The target audience for this educational initiative is neurologists, pediatricians, primary care physicians, and advanced practice providers.

Program Overview
Spinal muscular atrophy (SMA) is an inherited condition that places a tremendous burden on patients, families, caregivers, and healthcare providers, with patients experiencing a wide range of complications and symptoms that are detrimental to day-to-day life. Although SMA management has historically consisted of supportive care, the therapeutic landscape has changed dramatically following the recent approval of transformative medications, particularly disease-modifying therapies (DMTs) that improve motor function and survival. In this educational activity, experts in SMA seek to bring clinicians up-to-date by discussing recent advances in screening for SMA, as well as need-to-know information on newer DMTs, such as relevant clinical efficacy and safety data and recommendations for treatment selection.

Learning Objectives
Upon completion of this activity, participants should be better able to:

RECOGNIZE SMA based on signs, symptoms, and results of genetic tests
DESCRIBE safety and efficacy data from clinical trials evaluating disease-modifying therapies for SMA
SELECT an appropriate disease-modifying therapy approach for patients with SMA