New ERT For Late-onset Pompe Disease Shows Promise
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
2020 CAP Annual General Meeting
This year’s annual general meeting will be held on June 14, 2020, at 4pm EST.
Portraits of Pompe
This year, for International Pompe Day, we’re launching Portraits of Pompe, an ongoing curation of stories and photos from Pompe patients and families.
Sanofi Genzyme COVID-19 Update
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.
Amicus Therapeutics’ Clinical Trials During the COVID-19 Outbreak
Patients enrolled in all clinical studies for AT-GAA for Pompe disease, including the PROPEL pivotal study, will continue to receive study drug.
Amicus Therapeutics Provides Updates for AT-GAA for Pompe Disease
Amicus Therapeutics today announced clinical, regulatory and manufacturing advancements for AT-GAA.
IPA Update – Q4 2019
The latest newsletter from the International Pompe Association.
Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics
This development should provide Audentes with additional resources for their gene therapy programs, including a Phase 1/2 study for Pompe disease.
Audentes Therapeutics Update on Pompe and Other Programs
Audentes Therapeutics provides updates on their AAV-based genetic medicine program for serious rare neuromuscular diseases.
2019 CAP Annual General Meeting
The 2019 annual general meeting for Canadian Association of Pompe will be held by conference call on Sunday, May 26, 2019.