Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in ...
CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood ...
Rocket Pharmaceuticals announced yesterday it had reached agreement with the FDA on a potentially pivotal study of in vivo gene therapy candidate RP-A501. The drug candidate has faced a long journey ...
Get inside Wall Street with StreetInsider Premium. Claim your 1-week free trial here. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated ...