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Sarepta, gene therapy
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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 ...
This is the first time such results have been achieved in both children and adult patients born with a specific type of ...
Zacks Investment Research on MSN2d
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 ...
News Medical on MSN1d
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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