DMD manifests when the exons within a patient’s dystrophin gene are destroyed, resulting in the gene’s inability to fulfil ...

Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a dystrophin deficiency, which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch the ...

These PBGENE-DMD preclinical data compellingly demonstrate ... nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. This approach aims to restore the body’s native ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

Leerink Partners analysts pointed out in a note this morning that this 3.8-second improvement is greater than the 2.7-second benefit seen one year after taking Sarepta’s DMD gene therapy Elevidys.

This dystrophin gene correction approach which ... Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD ...

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 ...