Amicus Remains Hopeful about AT-GAA Despite Mixed Results from Phase 3 Study
Amicus Therapeutics recently announced topline results of its phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD).
Spark Therapeutics Has Begun a Phase 1/2 Gene Therapy Study for Late-Onset Pompe Disease
The Spark Therapeutics phase 1/2 gene therapy trial testing SPK-3006 to treat late onset Pompe disease has officially started with the first person being given their first (and theoretically only) dose.
Amicus Therapeutics Has Begun the FDA Submission for Their Pompe Drug, AT-GAA
Amicus Therapeutics has announced they have begun a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for their drug combination, AT-GAA, to treat late onset Pompe disease (LOPD).
New Review Article Highlights Latest Developments in Pompe Disease Research
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.
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.
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.