News from the World Muscle Society meeting


The World Muscle Society meeting was held in Saint Malo France in the first week of October and many companies shared results of their current Duchenne clinical trials. Here we highlight two news items.

Sarepta exon 53 skipping news

Sarepta Therapeutics presented results of their clinical trial of golodirsen (SRP-4053), a drug designed to skip exon 53 of the dystrophin Glossary Link gene, at the recent World Muscle Society Conference. This drug is for about eight percent of boys with Duchenne - those with a certain change in their Glossary Link DNA.

Golodirsen was tested in 25 boys with Duchenne in Europe for at least a year. Analysis of muscle biopsy samples from the trial showed about a 10-fold increase in dystrophin production which brought the level up to about one percent of normal dystrophin levels. Importantly, the new dystrophin Glossary Link protein was located in the correct place in muscle cells. Although this is still a small amount of dystrophin, it is thought that this could have a positive effect on preserving muscle function, at least to some degree.

This is the second exon skipping drug to show that this approach can increase dystrophin production in muslces, with Exondys 51 given FDA approval in September 2016. Another drug to skip exon 45 is also currently in clinical trial. Sarepta Therapeutics anticipates a meeting with the FDA regarding golodirsen in the first quarter of 2018.

Visit the Duchenne Deletion Tool on DuchenneConnect to help you understand if you/your child may be a candidate for an exon skipping therapy.

Catabasis phase 2 trial results

Catabasis have shared positive results from the Phase 2 MoveDMD open-label extension trial of edasalonexent in Duchenne.

Edasalonexent is designed to inhibit a protein in the muscle called NF-kB. It is thought that the increased levels of NF-kB, which are found in the muscles of boys with DMD, causes inflammation which further damages muscles. This also suppresses muscle stem cells that are required for muscle regeneration.

This latest update from Catabasis presents data from 36 weeks of treatment, which shows that progression of muscle weakness appears to slow down in treated boys. This data, although not conclusive, supports the initiation of a global phase 3 trial in the first half of 2018. This will include 4 to 7-year-old boys with Duchenne regardless of Glossary Link mutation type and who have not been on steroids for at least 6 months. Study sites outside of the US have not been announced yet.

Click here to read the press release and the most recent MoveDMD trial newsletter

Further information

-          More updates from WMS are available on the PPMD website

-          If you have any questions, please contact the DuchenneConnect Australia coordinators:

  • Phone: +61 (03) 8592 6431
  • Email: This email address is being protected from spambots. You need JavaScript enabled to view it.

Posted 10/25/2017