Jacqueline Glascock of Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy

Roche’s Evrysdi (risdiplam) is the market leader among the drugs for spinal muscular atrophy (SMA). Biogen’s Spinraza (nusinersen) has struggled, but company officials express confidence about future sales.

In a response to a survey, caregivers of people with spinal muscular atrophy identified the risk of severe adverse events and the need for permanent ventilation as the most important factors in treatment decisions. Access to treatment, including cost and availability, ranked third.

Some prior research had shown that gene therapy would not lower the amount of time children spend on ventilator support.

Two years after treatment, both girls show normal motor function and no signs of spinal muscular atrophy symptoms.

Apitegromab works by targeting a protein called myostatin that limits muscle growth and is designed to be used alongside existing spinal muscular atrophy (SMA) treatments.

The higher dose led to a more rapid decline in an important marker of neurodegeneration, according to a Biogen news release announcing the results.