While there is currently no cure for geographic atrophy, certain treatments, such as complement inhibitors, can help slow its progression by targeting immune system activity that damages the macula.

Biogen Inc. BIIB shares are up on Monday following the FDA’s approval of a new high-dose regimen for Spinraza (nusinersen), a ...

In a phase 2 trial, OCU410, a modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration, ...

The FDA has approved a higher-dose regimen of Spinraza for spinal muscular atrophy, providing a new treatment option for patients. The regimen includes 50 milligram per 5 milliliter and 28 milligram ...

Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Biogen today announced FDA approval of the High Dose regimen of SPINRAZA(TM) ...

A first-in-human clinical trial evaluating an investigational gene therapy targeting complement-mediated pathways in ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of ...

Scholar Rock Holding has resubmitted its application for its spinal muscular atrophy treatment to the Food and Drug Administration. The biopharmaceutical company said that it has sent in a new ...

Campaigners hope pilot will result in heel prick test for rare condition being approved across Britain ...