News
MedPage Today on MSN3d
Duchenne Gene Therapy Will Undergo Changes After Patient DeathsAt the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced a strategic restructuring plan ...
Sarepta Therapeutics, Inc. faces setbacks with Elevidys, safety issues, and workforce cuts, raising concerns about its ...
Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...
As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...
News Medical on MSN3d
Groundbreaking study offers a novel approach to enhance neuromuscular function in patients with Duchenne muscular dystrophyNew research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback