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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 ...
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 ...
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 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 ...
An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...
Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
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New UAB-developed gene therapy for ALS receives orphan drug designationThe U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...
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Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIAUltragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...
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