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Gene Therapy, Sarepta

Duchenne Gene Therapy Will Undergo Changes After Patient Deaths

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy, will carry a black box warning for acute liver injury and acute liver failure, drugmaker Sarepta Therapeutics said.

Bloomberg on MSN · 2h

Sarepta Says Another Patient Died After Receiving 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.

The Business Journals · 11h

Sarepta Therapeutics to lay off one-third of its workforce after gene therapy setback

Sarepta Therapeutics is facing a challenging period after the deaths of two patients who received its Columbus-invented gene therapy for Duchenne muscular dystrophy. Sarepta opens Columbus R&D center,

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 ...

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 ...

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 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 ...

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 ...

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 Executive16h

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 ...

Scientific American3y

We Need to Ground Truth Assumptions about Gene Therapy

Gene therapy’s rise to prominence has come with an extraordinarily high price tag. Novartis’s newly approved gene therapy, a one-time treatment for spinal muscular atrophy, is now the world ...

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