On-track to initiate IND-enabling activities for Wilson’s Disease program in 4Q 2024, with IND or CTA filing expected in 1H 2026 --CAMBRIDGE, ...

A low-carbohydrate diet may help adults with type 2 diabetes gain better blood sugar control and make it possible to decrease ...

On-track to initiate IND-enabling activities for Wilson’s Disease program in 4Q 2024, with IND or CTA filing expected in 1H 2026 -- CAMBRIDGE, Mass., Oct. 15, 2024 (GLOBE NEWSWIRE ...

GemmaBio and a related company, Franklin Biolabs, were spun out of the University of Pennsylvania's Gene Therapy Program on ...

Wilson disease is a genetic disorder where the body is unable to properly eliminate excess copper, leading to its buildup in organs like the liver, brain and eyes. This can cause liver damage ...

Ultragenyx Pharmaceutical RARE reported positive updates from the Stage 1 cohorts of its phase I/II/III Cyprus2+ study ...

Adapted from Schilsky ML, Tavil AS. Wilson disease. In: Schiff's Diseases of the Liver. Philadelphia: Lippincott Williams and Wilkins; 2006:1023–1040. Most practitioners prefer start with trientine.

Gemma Biotherapeutics, one of two companies launched by former University of Pennsylvania gene therapy program director Dr.

A minority of patients taking Ultragenyx Pharmaceutical’s Wilson disease gene therapy UX701 have come off standard-of-care drugs, leading the biotech to enroll a new cohort on a tweaked regimen ...