Families’ approach to finding a cure for CMD

Cure CMD’s solution is based on the direct experiences of three families affected by Congenital Muscular Dystrophy (CMD), a group of rare genetic disorders that cause muscle weakness from birth and can lead to a range of other physical and neurological impairments. CMD results from mutations in one of more than 30 genes, which cause muscles to deteriorate faster than the body can repair them.

The organization’s main objective is to accelerate research for treatments and improve care for individuals with CMD. To date, they have raised over $4 million for research, launched two clinical trials, and supported studies on the progression of the disease. One of their key initiatives is the Congenital Muscle Disease International Registry, which now includes over 3,500 participants. This registry provides important data for CMD research and facilitates collaboration among scientists, clinicians, and families.

Cure CMD places CMD patients at the forefront of its work. The current Executive Director, Rachel Alvarez, is a CMD patient herself, and many others within the organization also live with the condition. This ensures that the perspectives and needs of those directly affected by CMD shape the organization’s approach.

Beyond research, Cure CMD provides educational resources, connects families with specialized care, and advocates for better diagnosis and treatment. They rely on donations, grants, and partnerships with other organizations to fund their activities. The organization also aims to support the next generation of CMD researchers and expand its community outreach. Their work has led to a significant increase in community engagement and awareness, helping to address the practical needs of those living with CMD and supporting ongoing efforts in research and care.

For more information:
https://www.curecmd.org/
https://www.youtube.com/watch?v=reeRndgWZNA