STAT Plus: FDA approves first oral treatment for a rare muscle-destroying disease
The Food and Drug Administration on Friday approved the first oral therapy for spinal muscular atrophy, a rare and often devastating disease, adding a new option for patients who now rely on costly injected treatments.
The drug, from partners Genentech and PTC Therapeutics, is approved for adults and children with SMA, regardless of the severity of their disease. The once-a-day treatment will compete with a one-time gene therapy from Novartis and an injectable drug from Biogen that is administered every four months.
Continue to STAT Plus to read the full story…
