About Us

The Foundation to Eradicate Duchenne raises about $500,000 per year to support translational research in DMD.  A major goal is ‘seed funding’ for key infrastructure developments that would be expected to have a major impact on facilitating therapeutic advances worldwide.

Examples of previous seed funding efforts that have continued to become high-impact translational projects include:

• Cooperative International Neuromuscular Research Group (CINRG). FED recognized that an international academic clinical trial network was needed to facilitate DMD therapeutic research.  The establishment of CINRG was a major focus of FED and related Department of Defense CDMRP funding beginning in 2001.  CINRG has carried out the largest natural history study of DMD (CINRG DNHS), and this data is currently used to benchmark effects of drugs in initial open label trials by multiple pharmaceutical companies.
• Mdx mouse testing methods and consensus standard operating procedures. FED hosted the first international workshop to discuss consensus SOPs for measurement of the therapeutic effects of experimental drugs in the mdx mouse model of DMD.  This process was then carried forward collaboratively between TREAT-NMD and Children’s National Medical Center, with support from FED.  FED supported the initiation of a centralized mdx testing facility at Children’s National Medical Center, that then out-grew the academic setting and was spun off to AGADA BioSciences in 2013.
• Exon-skipping drug development. FED was an initial support of research into exon-skipping drug development, including support of IND-enabling toxicity studies via the DoD CDMRP, and proof-of-concept studies in DMD dogs  leading to the first clinical trials of exon-skipping using morpholino chemistries in DMD boys.  This work has culminated in the filing of a FDA NDA for NS Pharma exon 53 therapies.
• Vamorolone drug development. FED worked with DoD CDMRP program (see Page 45) to support the understanding of corticosteroid efficacy and safety in DMD; this work culminated in the development of the first-in-class small molecular drug vamorolone (VBP15).  Vamorolone clinical trials in DMD, carried out by the CINRG network, have shown efficacy in DMD open label trials, and a pivotal Phase III trial is expected to be completed in 2020.
• Mobile health outcomes development. Participation in clinical trials is critical to advance therapeutic approaches to DMD, but trials are a significant burden on the patients, and their families.  One emerging approach to lessen the burden on families, while providing ‘real world’ data on how the DMD patient is doing in his community, is through mobile health measures (wrist watches or other devices that monitor activity and health in ‘real time’).  Roxanna Bendixen, an Occupational Therapist at University of Pittsburgh is spearheading efforts to use the ActiGraph wrist monitor in measures of sleep, and of vamorolone in clinical trials.

Projects supported by FED: