The U.S. health regulator approved Marinus Pharmaceuticals Inc.’s lead drug to treat seizures associated with a rare genetic disorder in patients two years of age and older, the company said on March 18, sending its shares soaring around 36 percent.
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.