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At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
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Rocket Pharmaceuticals Shares Jump After FDA Grants Accelerated Review Status to Gene TherapyRocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...
The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from ...
CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
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 ...
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 ...
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