Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...
At the moment the only approved therapies are exon-skipping or stop codon read-through drugs, designed to restore dystrophin function in DMD patients with specific mutations in the dystrophin gene.
Secondary outcomes included assessments of dystrophin expression and motor function. At 12 weeks, patients treated with ...
Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...
Micro-dystrophin expression was maintained ... MRI assessments suggested the therapy stabilized or lessened muscle function deterioration, although the substudy was not powered for statistical ...
Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...
The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...
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