AUG 19 2026
Ultragenyx Pharmaceutical Inc. — Genglycos (pariglasgene brecaparvovec-opnr) $RARE
for glycogen storage disease type Ia
About
Glycogen storage disease type Ia is a serious metabolic disorder where the liver cannot properly release stored sugar into the bloodstream, leading to life-threatening low blood sugar and long-term liver damage. Genglycos is a gene therapy designed to deliver a working copy of the G6PC gene directly into liver cells. This one-time treatment aims to restore the liver's ability to manage blood sugar, potentially reducing the constant risk of dangerous hypoglycemia.
Drug Watch — recent news on Genglycos
- RARE Stock Gains on FDA Nod for Rare Disease Gene Therapy Genglycos - Yahoo Finance
- FDA grants accelerated approval to Ultragenyx’s Genglycos gene therapy - Yahoo
- Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy for GSDIa - Quiver Quantitative
- Ultragenyx Pharmaceutical Receives Accelerated US FDA Approval for Genglycos - Yahoo Finance
Sponsor Watch — recent news on Ultragenyx Pharmaceutical Inc.
- RARE Stock Gains on FDA Nod for Rare Disease Gene Therapy Genglycos
- Ultragenyx Wins FDA Approval for GENGLYCOS, Its First Gene Therapy
- Ultragenyx Announces U.S. FDA Approval of GENGLYCOS™ Gene Therapy, the First-Ever FDA-Approved Treatment Designed to Treat the Underlying Cause of Glycogen Storage Disease Type Ia (GSDIa)