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.
New Patients and Families
Welcome to the wonderful world of rare disease. It’s a much different world than it was a few years ago. There’s so much information out there, and there are places to turn for support and friendship. With social media, especially Facebook and Twitter, there are answers.
New Brunswick, 2014
Out of a stormy cloud comes a whole bunch o’ crazy people waving their hands… at the 2014 Fredericton Walk for Muscular Dystrophy. We were fortunate to gather in New Brunswick for our 2014 CAP Conference and were humbled to be presented with the Kaitlyn Hatchard Award with Annette Sebey.