News

InvestorsHub on MSN14h
Rocket Pharmaceuticals Shares Jump After FDA Grants Accelerated Review Status to Gene Therapy
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...
Managed Healthcare Executive1d
CMS Launches Value-Based Gene Therapy Program for Sickle Cell Disease
CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...
BioSpace1d
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 ...
Fierce Healthcare2d
CMS officially names participants in Cell and Gene Therapy Access Model
The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...
Researchers used gene therapy to restore hearing in adults
Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.
Managed Healthcare Executive17h
Applying Evidence Standards to Guide Gene Therapy Use in BCG-Unresponsive NMIBC
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 ...
US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
Live Science6d
In a first, congenital deafness in teens and adults treated with new gene therapy
This is the first time such results have been achieved in both children and adult patients born with a specific type of ...