Action Duchenne supported the preclinical funding and Phase 1 clinical trials of this innovative steroid-like intervention on Duchenne muscular dystrophy. 

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Eric Hoffman Ph.D., CEO of ReveraGen said:

“In summary, the support from Action Duchenne has been critical in moving the biomarker programme forward in the vamorolone (VBP15) clinical program in DMD.  With Action Duchenne help, we believe the integration of biomarkers is among the most robust and state-of-the-art of any pediatric drug development programme.”

VISION-DMD is an EC funded project undertaking Phase 2 clinical studies on vamorolone, a new treatment for Duchenne Muscular Dystrophy (DMD). The project was launched in January 2016 and aims to advance the clinical development of vamorolone (also known as VBP15) a dissociative steroid designed to have the efficacy of a steroid, without the severe associated side effects.

The project has key innovations for future clinical relevance, utilising exploratory pharmacodynamic biomarkers for clinical safety and clinical efficacy, developing novel MRI techniques to assess change in muscle, a mobile health band technology study, and the development of a mobile app providing a child friendly way to access patient information about the clinical trial. The business model for the development of vamorolone is through venture philanthropy. ReveraGen Biopharma Inc, the drug developer, has accessed grant funding and partnered with stake holder foundations to finance and to carry out the complex process of developing vamorolone for the treatment of DMD.

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