The executive director of Cure CMD discussed efforts being done to bring the stakeholder community together and improve the care for patients with congenital muscular dystrophies. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
"I think we’re all in for the cure, but the word ‘cure’ has a lot of baggage associated with it. We’re focusing on treatments and what’s perhaps more possible in a shorter term, and making sure that everybody is clear on what to expect at the various stages and ages for someone with CMD."
For years, Cure CMD has brought the clinical and patient community together to advance research and care for patients with congenital muscular dystrophies (CMDs), a group of diseases that cause muscle weakness at birth. Beginning on January 28, 2023, the organization is launching its Research Roundtable (https://www.curecmd.org/2023-research-roundtable), a 10-month program for the stakeholder community to share and learn about science, current and potential future research, and agree on attainable, realistic treatment goals.
From January to November, the program will include 21 meetings, each containing an hour’s worth of programming, with reading assignments, surveys, and online discussions for participants to complete between meetings. The program focuses on the 5 primary subtypes of CMD: collagen 6, the alpha-Dystrogycanopathies, LAMA2, LMNA, and SELENON. Affected individuals looking to participate will be required to attend at least 4 of 6 live virtual meetings, and are fully registered in the Congenital Muscle Disease International Registry (CMDIR).
In a recent interview with NeurologyLive®, Rachel Alvarez, executive director of Cure CMD, gave insight on the goals of the program, and how the organization plans to work towards them. Alvarez, who was diagnosed at an early age with an unspecified CMD and later diagnosed with COL6-RD, discussed who may benefit most from this type of program, and the stressed need to gain a better understanding of all aspects of CMD care and research.