Cure CMD is proud to partner with the Muscular Dystrophy Association and Cure ADSSL1 to support groundbreaking research at the Beggs Laboratory at Boston Children’s Hospital. This collaborative project will use next-generation genomic tools and AAV-based gene therapies to better understand disease mechanisms and advance potential treatments for SELENON-Related Myopathy, demonstrating how partnership across the rare disease community can accelerate progress toward meaningful t

Cure CMD joined rare disease advocates in Washington, DC for Rare Disease Week 2026, meeting with lawmakers to raise awareness of congenital muscular dystrophy and the urgent need for research support.

Researchers at the University of Basel are developing a gene therapy that could potentially treat those living with LAMA2-Related Dystrophy.

It Started With an Idea I’m Angela, and I live with SELENON-Related Myopathy. As a member of the CMD community, I have had the privilege of being connected to others living with similar neuromuscular diseases. Being part of this community has continually reminded me that while CMD can feel isolating at times, we are never truly alone. In 2017, I attended an MDA-sponsored premiere of I’ll Push You , a documentary that follows two best friends, Justin and Patrick. Justin has a

While we are thrilled to share a recent press release from Modalis Therapuetics , it’s important to keep in mind that the information provided includes “forward‑looking statements.” That means the company is sharing what they hope or plan to do, but these steps are not guaranteed and can change. The scientific paper mentioned in the press release describes years of careful testing of Modalis’ experimental therapy for LAMA2 (called MDL‑101) in animal models. Publishing this w

The MAGIC consortium held its annual General Assembly from 28–30 October in Lisbon, hosted by the Gulbenkian Institute for Molecular Medicine (GIMM). Consortium members from around the world came together to share progress and plan next steps in this project that aims to accelerate the development of new treatments for rare neuromuscular conditions. This meeting was a powerful reminder that the building of global networks consisting of researchers, clinicians, and advocates,

We're thrilled to share that after several years following Cure CMD's submission to the Social Security Administration requesting the addition of LMNA-CMD to the Compassionate Allowance List, this CMD subtype is now included. The CAL list is SSA's way of fast-tracking help for people with the most severe medical diagnoses. Conditions on this list are considered serious enough to qualify for disability benefits and your application will be moved to the front of the line. Speci

In August, seven CMD advocates from Wisconsin, Ohio, and New York joined Rare Across America, connecting with Members of Congress to spotlight critical issues affecting the rare disease community. Discussions focused on restoring the Rare Pediatric Disease PRV Program, improving Medicaid access for kids needing out-of-state care, and protecting NIH and FDA research funding. Advocates shared personal stories and called for bipartisan support to ensure progress in treatment dev

Over 340 attendees gathered in Philadelphia for the 2025 Scientific & Family Conference, uniting families, clinicians, and researchers to share stories, advance care, and spark new collaborations. Highlights included 60 sessions, Kids Camp, young adult meetups, and the Scientific Symposium.

Cure CMD’s Creative Contest celebrates its 5th year of providing an outlet for people living with CMD and other neuromuscular conditions to promote the creative expressions of their stories through visual, physical, or performance art.

Researchers at the Wright Lab at OHSU’s Vollum Institute have discovered that the protein Dystroglycan is vital for brain connectivity, revealing a key neurological role in Dystroglycanopathy and opening new pathways for CMD research and treatment.

The FDA’s first Platform Technology Designation marks a major step forward for rare disease treatments, allowing reuse of key therapy components across related conditions. This breakthrough could accelerate CMD therapies, but Cure CMD needs your support to keep the momentum going.