ReveraGen BioPharma has announced encouraging preliminary results of their early stage clinical trial of Vamorolone, a potential drug that researchers hope could offer similar benefits to steroids with reduced side effects.

The trial took place across 6 countries including the UK and evaluated 4 doses of vamorolone in 48 boys aged 4-7 who are living with Duchenne. After receiving the potential drug for 2 weeks, the boys entered an extension of the trial, where they continued to receive Vamorolone for a further 6 months.

Researchers used multiple functional tests of strength and endurance to test the effectiveness of Vamorolone and compared the results to natural history data from boys who were not treated. Encouragingly, those who received Vamorolone showed dose-related benefits similar to those seen in the historic CINRG trial of prednisone (a traditional corticosteroid).

Action Duchenne played a key role in setting up the clinical trial and a research project we funded developed a test that allowed researchers to monitor the level of Vamorolone in trial participants using biomarkers.

The side effects of anti-inflammatory steroids often detract from patient quality of life, particularly in children. The potential of vamorolone to reduce this burden may improve the quality of life of children with DMD and their families. -Prof. Eric Hoffman, Binghamton University and CEO of ReveraGen BioPharma

Although Vamorolone binds to the same proteins on a cell as the traditional glucocorticoid steroid, it separates the anti-inflammatory actions from those that cause safety concerns, so Vamorolone could offer the benefits of steroids with reduced side effects. Further, larger trials are now underway to test whether Vamorolone is safe and effective and we will keep you updated as we find out more.

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