duchenne muscular dystrophy

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Sarepta, Duchenne and gene therapy

2nd death reported in Sarepta gene therapy trial for Duchenne muscular dystrophy

Sarepta Therapeutics (NASDAQ:SRPT) reported a second fatality from acute liver failure in a patient treated with its gene therapy, Elevidys, designed for Duchenne muscular dystrophy (DMD), Bloomberg News reported Sunday.

STAT · 1d

Sarepta reports second patient death after treatment with Duchenne gene therapy

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death of a second recipient.

The American Journal of Manage… · 2d

DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD).

EconoTimes1h

Roche Halts Elevidys Dosing in Some Duchenne Patients After Fatalities

Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...

19m

Sarepta Shares Dive on Second Fatal Liver Failure Tied to Elevidys

Sarepta Therapeutics shares dived 42% in premarket trading after the company disclosed a second reported case of acute liver failure that resulted in a death tied to its Duchenne muscular dystrophy ...

2don MSN

Questions over cost of muscular dystrophy drug in Belfast Trust

The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

The American Journal of Managed Care3d

RGX-202 Gene Therapy Improves DMD Trajectory, Biomarkers

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

RTÉ Ireland4d

Families call for approval of new 'game changer' Muscular Dystrophy drug

Parents of children with an incurable muscle-wasting condition have called on the Minister for Health to accelerate the ...

New hope for Deal family of nine-year-old boy with Duchenne muscular dystrophy

The parents of a nine-year-old boy battling a devastating muscle-wasting condition say new research could offer them more ...

Managed Healthcare Executive12d

Encouraging Results from Phase 1 Trials, Says Satellos Chief Scientific Officer. Now It's On to Phase 2

Satellos Bioscience Chief Scientific Officer discusses early-stage trials of the company's Duchenne muscular dystrophy drug, ...

Capricor Therapeutics Announces Key Regulatory Updates for its Duchenne Muscular Dystrophy Program

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support ...

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