News

PMLive2h
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 ...
EconoTimes4h
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 ...
STAT54m
Pharmalittle: We’re reading about a Sarepta setback with a DMD drug, FDA workload causes a delay, and more
In today's Pharmalittle roundup, we're reading about a Sarepta setback with its Duchenne drug, the FDA workload causing a ...
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 ...
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, ...
Hosted on MSN13d
Cardiac Complications of Duchenne Muscular Dystrophy
Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies ...
RTÉ Ireland5d
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 ...
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 ...