This year, for International Pompe Day, we’re launching Portraits of Pompe, an ongoing curation of stories and photos from Pompe patients and families.
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.
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 today announced clinical, regulatory and manufacturing advancements for AT-GAA.
The latest newsletter from the International Pompe Association.
This development should provide Audentes with additional resources for their gene therapy programs, including a Phase 1/2 study for Pompe disease.
Audentes Therapeutics provides updates on their AAV-based genetic medicine program for serious rare neuromuscular diseases.
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.
Located in Philadelphia, PA, Amicus Therapeutics is establishing a new Global Research and Gene Therapy Center of Excellence.