CDKL5 deficiency disorder (CDD) Archives

U.S. FDA approves Marinus Pharma’s drug to treat seizures; shares jump

Business, CDKL5 deficiency disorder (CDD), Drug Enforcement Administration (DEA), Drug Pricing, FDA, Rare Genetic Diseases, Seizures, Shares, Therapeutics

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.

March 18, 2022/by Reuters Health

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