Dr John Brandsema Highlights Progress in SMA Treatment and Improved Outcomes for Patients

The introduction of 3 FDA-approved therapies for spinal muscular atrophy is enabling clinicians to treat the disease completely different from how it was handled in the past.

Prior to 2016, the only options available to patients diagnosed with spinal muscular atrophy (SMA) was best supportive care. However, the introduction of 3 FDA-approved therapies—nusinersen in 2016, onasemnogene abeparvovec in 2019, and risdiplam in 2020—has changed the treatment paradigm.

Now, patients are being identified screened earlier, diagnosed sooner, and treated faster to alter the disease trajectory, keep patients stable, and improve outcomes for patients with SMA, explained John Brandsema, MD, neuromuscular section head, Children’s Hospital of Philadelphia.

During an interview with The American Journal of Managed Care® (AJMC®), Brandsema reviewed the treatment landscape, the importance of diagnosing SMA early, and discussions with families and patients when considering the 3 therapies.