DMD Diamond: Unleashing Next-Gen Blockchain with Cooperative HBBFT
SAN FRANCISCO, CA / ACCESS Newswire / March 26, 2025 / As the blockchain landscape evolves towards interconnected, heterogeneous multi-chain ...
Nature10y
Sex-linked Diseases: the Case of Duchenne Muscular Dystrophy (DMD)
Scientists have been searching for ways to cure DMD for as long as they have known about the disease. Today, based on the wealth of knowledge about the underlying genetic and molecular mechanisms ...
Neurology Advisor2d
Patient Dies Following Treatment With DMD Gene Therapy Elevidys
Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.
The American Journal of Managed Care5d
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort ...
GEN - Genetic Engineering and Biotechnology News12d
DMD Patient Dies After Treatment with Sarepta Gene Therapy
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
Monthly Prescribing Reference26d
Deramiocel Granted Priority Review for DMD Cardiomyopathy
In male patients with DMD, treatment with deramiocel reduced upper limb function deterioration compared with placebo. The Food and Drug Administration (FDA) has granted Priority Review to the ...
The Pharma Letter3d
Wave Life releases positive Phase II results for WVE-N531 in DMD
Cambridge, USA-based RNS specialist Wave Life Sciences (Nasdaq: WVE) has announced positive data from the Phase II FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being ...
The Pharma Letter12d
Italfarmaco and Medis ink distribution deal for DMD drug
Privately-held Italian pharma company Italfarmaco and Slovenia-based Medis Therapeutics have announced an exclusive ...