Pompe disease Archives

Phase III Trial Failure for Pompe Disease Drug

Analysts, Clinical Data, Clinical Trials, Double-Combination Therapies, Inherited Lysosomal Storage Disorders, Pompe disease, Pompe disease, Primary Endpoints, R&D, Recombinant human acid alpha-glucosidase (rhGAA) enzym

Amicus Therapeutics announced topline results from the Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease.

February 12, 2021/by Andrew Humphreys

Amicus and Penn Broaden Gene Therapy Collaboration

Adeno-Associated Virus (AAV), Angelman Syndrome, Business, CDKL5 deficiency disorder (CDD), Collaborations, Fabry disease, Gene Therapy, Gene Transfer Technologies, Inherited Lysosomal Storage Disorders, Muscular Dystrophy, Mycopolysaccharidosis Type IIIA (MPS IIIA), Mycopolysaccharidosis Type IIIB (MPS IIIB), Myotonic Dystrophy, Niemann-Pick Type C (NPC), Pompe disease, Rett Syndrome, University of Pennsylvania (Penn)

Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.

May 29, 2019/by BioSpace