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

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

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

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 ...

Exons are coding sections of an RNA transcript, or the DNA encoding it, that are translated into protein. Exons can be separated by intervening sections of DNA that do not code for proteins, known ...

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

Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD Date and Time ...

Shares of Wave Life Sciences Ltd . (NASDAQ: WVE) rose 14% today, following the company’s announcement of positive results from its FORWARD-53 clinical trial in Duchenne muscular dystrophy (DMD), 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 patients than exon skippers. Key findings from the ...