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Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...
CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...
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 ...