News

Cyclists to arrive in Bar Harbor after cross-country ride to raise awareness for rare disorder
Cyclists led by Charlie Nuck will arrive in Bar Harbor Saturday to raise awareness and funds for the rare neurogenetic ...
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and ...