An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
Persons with genetic conditions cannot be asked by their employers, or any other agency, to disclose that information.
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.
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.