by CAP | Feb 15, 2021 | News
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).
by CAP | Feb 10, 2021 | Talk Pompe
Cheryl Wong Po Foo, PhD, Director of Clinical Development, Audentes Therapeutics, responsible for the advancement of the clinical development program for Pompe disease.
by Victoria Radke | Feb 1, 2021 | News
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.
by CAP | Dec 3, 2020 | News
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).
by CAP | Sep 20, 2020 | News, Research & Studies
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.
by CAP | Jul 12, 2020 | News, Research & Studies
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.
by CAP | Mar 28, 2020 | COVID-19
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.
by CAP | Mar 19, 2020 | COVID-19
Patients enrolled in all clinical studies for AT-GAA for Pompe disease, including the PROPEL pivotal study, will continue to receive study drug.
by Brad Crittenden | Jan 7, 2020 | News
Amicus Therapeutics today announced clinical, regulatory and manufacturing advancements for AT-GAA.
by Brad Crittenden | Feb 25, 2019 | News
Amicus Therapeutics announced today that they have received Breakthrough Therapy Designation (BTD) for their AT-GAA treatment for Pompe disease.