Duchenne Muscular Dystrophy

ReveraGen co-founder Dr. Hoffman identified dystrophin deficiency as the cause of Duchenne muscular dystrophy in 1987, and has worked to translate advances in the laboratory to patient and family care through molecular diagnostics, and increased understanding of the disease process in Duchenne muscular dystrophy. Drs. Hoffman and Nagaraju worked to define inflammation through NFkB pathways as the earliest manifestation of muscle pathology in DMD, present soon after birth. Drs. McCall, Nagaraju and Hoffman then worked to identify vamorolone (VBP15) as an inhibitor of muscle inflammation, and this became the scientific basis for initiation of the vamorolone clinical trials and investigational drug regulatory program in DMD.

As noted above, Duchenne muscular dystrophy is caused by loss of the dystrophin protein in patient muscle. This initiates a series of cellular and tissue responses that eventually lead to fibrofatty replacement of skeletal muscle, and deterioration of muscle function. Vamorolone was developed with the hope that its targeting effect may slow the initial inflammatory reactions of skeletal muscle to dystrophin deficiency and aims to slow the downstream fibrofatty replacement.