News

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
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For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
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Sarepta Reports a Third Gene-Therapy Related Patient Death

The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute ...

Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
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miR-33 inhibition as a novel therapeutic approach for treating muscular dystrophy

MicroRNA-33a and microRNA-33b are encoded within the mammalian SREBF1 and SRBF2 genes, respectively, and both SREBF1 and ...
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SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program

Sarepta plummets after a third death in its muscular dystrophy program for investigational gene therapies, prompts FDA action and intensifies safety scrutiny.

Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...

Children's Hospital LA pauses use of Sarepta gene therapy for all patients

Children's Hospital Los Angeles, citing recent U.S. Food and Drug Administration actions, on Monday said it has paused usage of Sarepta Therapeutics' gene therapy Elevidys in all patients with ...

Drugmaker Refuses F.D.A. Request to Pull Treatment Linked to Patient Deaths

The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Elevidys, after three patients died from ...

Sarepta tells FDA it won’t halt shipments despite patient deaths

In a highly unusual move, Cambridge-based Sarepta said late Friday won’t comply with a request from the Food and Drug ...