Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for Pompe disease.
Health Canada has approved avalglucosidase alfa (Nexviazyme™) for the long-term treatment of late-onset Pompe disease in patients over the age of 6 months.
In a recent announcement, the Pompe disease community learned that the U.S. Food and Drug Administration (FDA) has approved Nexviazyme® for the treatment of late-onset Pompe patients that are one year old and older.
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).
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.
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.