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

Some muscles are resistant to the loss of motor neurons seen in patients with spinal muscular atrophy, and new research has discovered that even in muscles that appeared resistant to SMA, subtle changes had occurred at the cellular level.

The combination of Spinraza and Zolgensma could improve clinical outcomes in infants with spinal muscular atrophy without added risks.

Preserving the ability to swallow reduces the need for invasive interventions and lowers the risk of life-threatening complications such as aspiration pneumonia.

In a meta-analysis of 57 studies, Zolgensma achieved a 95% survival rate and reduced the number of patients requiring ventilatory support.

The study drew on data from the RESTORE registry for 34 patients with tracheostomies who received Zolgensma (onasemnogene abeparvovec).

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.