FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

A higher dose of Spinraza (nusinersen) may be more effective than the currently approved dosing schedule in people with spinal muscular atrophy (SMA) for maintaining motor function, according to data ...

While 3 US Food and Drug Administration (FDA)-approved drugs are available for the treatment of spinal muscular atrophy (SMA), managing the disease requires a multidisciplinary team and approach. SMA ...

treatment with OAV101 IT demonstrated stabilization of motor function over 52 weeks of follow-up in patients who had previously discontinued treatment with Spinraza (nusinersen) or Roche’s RHHBY ...

Key to that mission is the “STEER” study, a late-stage trial of children with moderate SMA who are older than two years and have never before received certain targeted medicines like Zolgensma, Biogen ...

The STRENGTH study found OAV101 IT stabilized motor function in patients who had discontinued Spinraza or Evrysdi ... who had discontinued treatment with nusinersen or risdiplam.

tolerability and efficacy of OAV101 IT in patients with SMA aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam. Key findings: OAV101 IT demonstrated a ...

in individuals who had tried but discontinued treatment with Biogen’s Spinraza or Roche’s Evrysdi in the phase 3b Strength trial. Among those treatment-experienced SMA patients aged 2 to less ...

2 But this indication leaves out those older than 2 years, who are currently limited to non–gene therapy options like nusinersen (Spinraza; Biogen) and risdiplam (Evrysdi; Genentech), and in ...