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Sarepta, Gene Therapy

· 1h · on MSN

Sarepta Says Another Patient Has Died After Gene Therapy

Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company after the recent deaths of two teenage boys.

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Southwest Journal · 1h

Breaking: Second Patient Death Prompts FDA Investigation of Sarepta’s Elevidys Gene Therapy

pharmaphorum · 53m

Third death reported with a Sarepta gene therapy

Miltenyi Biotec and THSTI FIVE partner to launch India’s first hands-on CAR-T cell and gene therapy training curriculum

Our Bureau, Mumbai Friday, July 18, 2025, 17:45 Hrs [IST] ...

Fierce Pharma3d

Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

9don MSN

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 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., ...

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 ...

InvestorsHub on MSN22h

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 ...

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