CURE CMD

Based out of Torrance, CA, Cure CMD also holds events across the country and internationally to serve scientists and families seeking knowledge and information about Congenital Muscular Dystrophy.

The organization was founded in 2008 by three parents whose children were diagnosed with congenital muscular dystrophy and today, Cure CMD is a leading source of research news, support, and connections to CMD experts with a global network of 2,000+ affected children, adults, and family members.

Cure CMD has helped to initiate and continue numerous research projects since 2008, awarding more than $2M to leading scientists studying CMD. However, numerous gaps in knowledge about CMD still exist. Fortunately, with an initial natural history study complete, COL6-CMD is now the focus of a recently successful Stage 1 clinical trial that tested the compound Omigapil for safety in children and adolescents. Initial results are promising, and with other strategies including CRISPR/Cas9 gene editing and other management techniques on the horizon, CMD is one of the most exciting and fast-growing areas of rare disease research.