Latest News & Articles
Past News & Articles
Beam 301 -STRIVING FOR A NEW POTENTIAL THERAPY FOR GSD1a
THE BEAM-301 CLINICAL STUDY A phase 1/2, multicenter, open-label, single-ascending dose study to evaluate the safety and efficacy profiles of [...]
We Need Your Voice!
EL-PFDD interactive virtual meeting on GSD1a. Living with Glycogen Storage Disease Type 1a October 9, 2025 More Information [...]
Ultragenyx Announces Positive Longer-term Data from Phase 3 Study
Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage [...]
Introduction to Beam’s Clinical Development Program for GSD1a
View Presentation
World-First Gene Editing Trial for Rare Genetic Disease at UConn Health
UConn Health and Connecticut Children’s have made history by dosing the first patient in a clinical trial using gene [...]
Beam 301 Clinical Study Information for GSD 1A
Unleashing The Power Of Gene Editing To Potentially Transform The Lives Of People Living With Glycogen Storage Disease 1a [...]
