Telehealth services have been critical to maintaining safe and consistent access to health care for patients with rare diseases such as Congenital Muscular Dystrophy throughout the pandemic. The rare disease community recognized the advantages of telehealth even long before the pandemic hit in 2020. Telehealth provides an opportunity for patients to receive care from a disease-specific expert who practices in another part of the country. We know that traveling hundreds and sometimes thousands of miles for a routine medical appointment is not uncommon for an individual with a rare disease and their caregiver. Read More.

In December 2021, the FDA approved Cure CMD’s application to host an externally-led PFDD meeting for the five primary subtypes of congenital muscular dystrophy. This four-hour meeting on Friday, July 1 will take place at 2022 SciFam.

In the coming weeks,community members will be given the opportunity to provide their perspectives through the completion of a survey, comment submission, pre-recorded video testimonials, and the chance to contribute live at the PFDD meeting. Check out the agenda, and join us for this critical piece of the CMD drug development process. Read More.

Cure CMD is thrilled to announce a third year of funding from the Chan-Zuckerberg Initiative’s Rare As One Network, an effort to strengthen rare disease communities, connect them with one another, and build capacity to accelerate research. Along with a $150,000 grant for 2022 and the opportunity to network with others in the rare disease space, membership in the RAO cohort includes connections to partners, access to experts, and an extensive library of webinars. Read more. 

On the road toward treatments and for CMD, we’ve come a long, long way to learn about causes and complications. Sometimes, it can seem like the more we learn, the more there is to learn. A bit of good news in our journey toward more comprehensive knowledge is that a new project offers a new foundational understanding of our family of conditions: the Pediatric Cell Atlas of Skeletal Muscle. It’s an important step in building knowledge and subsequently, therapies for CMD and other neuromuscular conditions. Read more.

The hardest part was keeping it a secret - feeling the excitement overflowing inside, but not being able to tell friends at school. Read More.

“Expect her not to survive beyond age 1,” was what my young single mother was told in the first few months of my life when I wasn’t strong enough to suck from a bottle. Just before my first birthday, the prognosis was, “expect her to never walk.” After I achieved ambulation at 14 months, the prognosis was, “The oldest living patient we know with a condition like hers is age 27. Most die of pneumonia.” Read More.