We are pleased to share that Vamorolone, an alternative medicine to corticosteroids, has shown positive results in a Phase 2 Study.

Vamorolone was developed as a treatment for Duchenne muscular dystrophy, to offer the benefits of steroids, but without some of the side effects that can have a serious impact on patients’ quality of life.

The results from Santhera Pharmaceuticals and ReveraGen BioPharma showed that boys on Vamorolone did better than those taking a placebo. It also found that Vamorolone had a better safety profile than Prednisolone, one of the corticosteroids prescribed to Duchenne patients.

In 2014, the Duchenne Research Fund, Duchenne UK and Joining Jack helped fund the $2.1m Phase 1 study on Vamorolone. This funding allowed the company to raise further investment to fund the Phase 2 study. The trial, called VISION-DMD, is a Phase 2b trial looking at the efficacy, safety, pharmacokinetics (movement in the body) and pharmacodynamics (effects on the body) of Vamorolone in comparison to corticosteroids and placebo.

The Duchenne Research Fund also co-funded the post of Dr Michela Guglieri, the consultant who is running the trial in the UK.

Please click here to read the press release here.

Click here to learn more about Vamorolone.