The FDA approved the first therapy for glycogen storage disease type Ia in patients 8 and older
GSDIa forces constant fear of low blood sugar and lifelong dietary control. A first approved therapy means the disease is no longer something medicine could…
More context
How / context
Accelerated approval for Genglycos (pariglasgene brecaparvovec), a gene therapy for GSDIa.
Why it matters
GSDIa forces constant fear of low blood sugar and lifelong dietary control. A first approved therapy means the disease is no longer something medicine could only manage by hand.
Sources
- Primary · fda.gov ↗
- Feed · fda_press
