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.
Amicus Therapeutics Presents Positive Preclinical Pompe Disease Gene Therapy Data
In what could eventually be very good news for Pompe patients, Amicus Therapeutics presents positive data from their preclinical gene therapy program for Pompe disease.
Audentes Therapeutics’ “Be Bold for Courageous Patients” Program to Support Patients Living with Rare Diseases
In recognition of Rare Disease Day, Audentes Therapeutics announces their new “Be Bold for Courageous Patients” program.
Amicus Therapeutics establishes a 75,000 sq. ft. Gene Therapy Center of Excellence
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.