New Treatments for Rare Diseases: FDA Approvals and Trial Wins

FDA approves Scholar Rock's Isembyld, the first therapy targeting muscle loss in spinal muscular atrophy (SMA). A late-stage trial showed it improved young patients' motor skills when combined with existing treatments. Side effects: Potential concerns include unknown long-term effects of myostatin inhibition, though the trial showed a favorable safety profile.

Refills

Stories worth a second look:

1. Ionis Launches Zanvastro for Alexander Disease — The first...