Canadian Association for Glycogen Storage Disease

10/14/2025

Significant progress.

Thank you to our sponsor Beam Therapeutics for supporting the EL-PFDD ON GSD1a and for all your work to help us find a cure! Beam is currently enrolling adult GSD1a patients for their Phase 1/2 Clinical Trials to evaluate BEAM-301 for the potential treatment of GSD1a. There are 3 clinical study sites open across the U.S. Please contact [email protected] for more information.

10/13/2025

Significant event.

On October 9, 2025, The Children's Fund for GSD Research hosted an Externally Led Patient Focused Drug Development Meeting (EL-PFDD). This meeting gave patients and families an opportunity to share their voices and stories with the purpose of accelerating research and improving care.  

10/05/2025

Hi everyone! Our EL-PFDD meeting with the FDA is this Thursday. We hope you will tune in and participate! You can visit https://www.curegsd.org/pfdd to view the discussion questions and send comments in now. We will be reading your comments throughout the meeting (no identifying information) and they will also be included in the Voice of the Patient report which will summarize the meeting and live in the FDA archives indefinitely. Please share your perspective. We need your help to encourage future research and new treatment options for GSD1a.

03/12/2025

For information purposes..

Congratulations to Dr John Mitchell, Dr David Weinstein, Kathy Ross RD, et al. for publication of new study:
Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa)

Glycogen storage disease type Ia (GSDIa) is a rare, life-threatening, inherited carbohydrate metabolism disorder caused by glucose-6-phosphatase (G6Pase) deficiency, which is essential for glycogenol...

03/28/2023

Inviting you to check out our website..

02/28/2023

International Rare Disease Day
February 28