Clinical Trials Arena on MSN16h
Regenxbio’s DMD gene therapy drives 122.3% biomarker uptick in three-year-oldThe interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.
About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...
Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle mass, due to mutations in the dystrophin gene. Without the corresponding functional protein ...
Entrada Therapeutics announced Monday that the FDA lifted the clinical hold on its Duchenne muscular dystrophy drug, and it plans to start a Phase 1b … ...
If you’re living with Duchenne muscular dystrophy (DMD), planning ahead can help support the success of future education, employment, and housing efforts. Duchenne muscular dystrophy (DMD ...
TALAPRO-2 showed talazoparib and enzalutamide reduced death risk in mCRPC, effective across HRR gene alterations, highlighting the need for next-generation sequencing. STOPCAP meta-analysis found ...
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