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Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...
CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...
Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
This is the first time such results have been achieved in both children and adult patients born with a specific type of ...
Gene therapy’s rise to prominence has come with an extraordinarily high price tag. Novartis’s newly approved gene therapy, a one-time treatment for spinal muscular atrophy, is now the world ...
Since gene therapy was first proposed to treat such genetic diseases in the 1970s, it has had thrilling highs — including the first successful gene therapy, in 1990, in a 4-year-old born with ...
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...