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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 · 7h

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.

Seeking Alpha on MSN · 1h

Sarepta plunges after third patient death tied to gene therapy - report

Sarepta Therapeutics (SRPT) said a third patient has died from acute liver failure after receiving one of its gene therapies, intensifying scrutiny over the safety of its treatments, Bloomberg reported.

MedPage Today on MSN2d

CMS Rolls Out Value-Based Gene Therapy Model for Sickle Cell Disease

The model was initially launched by the Biden administration in February 2023, and CMS announced that SCD would be the first focus of the model the following January. This is the ...

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 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 ...

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.

6don MSN

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 ...

InvestorsHub on MSN19h

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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