New ERT For Late-onset Pompe Disease Shows Promise

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.

Amicus Therapeutics Announces Positive 18-Month Data in Pompe Disease Phase 1/2 Study

Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society

CRANBURY, N.J., Oct. 04, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced additional positive results from a global Phase 1/2 clinical study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) in cells.