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Sarepta Stops Shipments of Elevidys For Non-Ambulatory DMD Patients
A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...
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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 ...
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Lilly’s amylin candidate shows early promise
Supernus Pharmaceuticals said it is acquiring Sage Therapeutics, the distressed maker of a drug for postpartum depression, ...
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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 ...
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Cardiac Complications of Duchenne Muscular Dystrophy
Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies ...
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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 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 ...