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).
Cheryl Wong Po Foo, PhD, Director of Clinical Development, Audentes Therapeutics, responsible for the advancement of the clinical development program for 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 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).
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’s annual general meeting will be held on June 14, 2020, at 4pm EST.
“Set your goals high and work to achieve them; make the improbable possible!”
This year, for International Pompe Day, we’re launching Portraits of Pompe, an ongoing curation of stories and photos from Pompe patients and families.