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 ...
Histone deacetylase inhibitor. Givinostat (Duvyzat) is the first non-steroid drug approved to treat people with all genetic variations of DMD. It delays the disease's progression by targeting an ...
The drug’s mechanism of action ... the treatment of muscular dystrophies and other degenerative diseases. DMD remains an incurable condition, but breakthroughs like K884 offer a glimmer of ...
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...
The first successful phase II trial in Duchenne muscular dystrophy (DMD) heart disease rolled out from Cumberland Pharmaceuticals Inc., which said top-line findings from the experiment called Fight ...
An experimental gene therapy for Duchenne Muscular Dystrophy ... neuromuscular diseases constitute a bigger challenge given the absence of knowledge on the physiopathological mechanisms and ...
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
Our past work uncovered the JAG1 genomic locus as a modifier of DMD (Vieira et al ... to manipulate the zebrafish genome to discover novel pathways and mechanisms for normal and disease muscle ...
Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $ ...
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