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MedPage Today on MSN2d
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 ...
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 ...
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...
The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...
News-Medical.Net on MSN3d
Targeting GLUD1 shows promise in restoring muscle function in Duchenne muscular dystrophyNew research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...
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