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Sarepta Tags DMD Gene Therapy Elevidys With Black Box Warning, Axes 500 Staff
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.
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 ...
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.
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 ...
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
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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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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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