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A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...
Massachusetts-based Sarepta Therapeutics, facing scrutiny from regulators, is reducing its global workforce by 36% in an ...
Rocket Pharmaceuticals jumps 16.8% as FDA grants RMAT status to RP-A601 on positive phase I heart disease data.
In early research, OCU410ST, a gene therapy for Stargardt disease, demonstrated slowed lesion growth by 48% at the 12-month follow-up in evaluable treated eyes.
Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...