As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its ...

DMD mutations are particularly common in “hotspot” areas of the gene (exons 45–55 and 2–10). Because the genetic underpinnings of Duchenne are known, researchers can devise gene-editing fixes to the ...

The large DMD gene has 79 exons. For DMD patients with suitable mutations, exon skipping is one way to convert out-of-frame mRNA translation to a properly aligned reading frame. Oligonucleotides ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five Duchenne ...

Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads ... of a defective gene by delivering two ARCUS nucleases in a single AAV such as in the DMD program for oral presentation ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

Two-year data showed significant functional improvements in patients treated with delandistrogene moxeparvovec compared to controls, despite initial trial endpoint failure. The treated group ...

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 ...