duchenne muscular dystrophy

News

Sarepta Therapeutics, Duchenne and Gene

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.

Newsable Asianet News on MSN · 10h

Sarepta Pauses DMD Gene Therapy Trial After Second Death; Retail Traders Brush Off Risk

Retail investor chatter around Sarepta Therapeutics surged on Sunday after the company announced it had suspended shipments and paused a clinical trial of its gene therapy Elevidys for non-ambulatory patients with Duchenne muscular dystrophy following a second patient death from acute liver failure.

PMLive1h

Duchenne muscular dystrophy – diagnosis, clinical development and global research

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...

Sarepta Therapeutics Stock Plummets Over 40% On Second Fatal Case Linked To Its Gene Therapy

Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and ...

EconoTimes3h

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 ...

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 ...

3don 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, ...

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 ...

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 ...

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 ...

Medical Marketing & Media7d

How the Muscular Dystrophy Association celebrated 75 years of research

The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.

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 ...

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