The FDA has granted accelerated approval of a gene therapy to treat children and adults aged at least 8 years with glycogen storage disease type Ia, according to an industry press release. Pariglasgene brecaparvovec-opnr (Genglycos, Ultragenyx) is the first therapy approved by the FDA to reduce burden of care associated with glycogen storage disease type Ia, according to the company. Glycogen

The FDA has granted accelerated approval of a gene therapy to treat children and adults aged at least 8 years with glycogen storage disease type Ia, according to an industry press…

If at first you don’t succeed in gene therapy, then try, try again. | If at first you don’t succeed in gene therapy, then try, try again, is the lesson Ultragenyx likely took home…