Rare disease drugmaker Ultragenyx builds case for its gene therapy franchise with fresh trial updates
Focused on combating a smattering of rare diseases with an arsenal of biologics, small molecules, and gene therapies, Ultragenyx Pharmaceutical this week issued some updates on two of its experimental gene therapies as it works on building a franchise for one-time cures.
On Friday, the company unveiled early data on the effect of DTX401, its gene therapy under investigation, in patients with glycogen storage disease type Ia (GSDIa), a disease that affects roughly 6,000 people globally and is characterized by the inability to regulate blood sugar.
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