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

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

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,

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.

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.

From shaping care guidelines in the Netherlands to tackling gene therapy challenges at the MDA Summit, Cure CMD is driving progress in research and care. Join us at the 2025 SciFam Conference this August in Philadelphia—registration is open!

CMD Community Member, Justin Moy, shares his journey with LAMA2-related muscular dystrophy and his life quest to cure his own condition.

Modalis Therapeutics program for LAMA2-RD gene therapy granted Rare Pediatric Disease Designation by FDA.

We are honored to support groundbreaking discoveries related to LMNA-CMD.

We are thrilled to announce a new clinical workshop focused on establishing CMD subtype-specific care recommendations.

We are honored to support groundbreaking discoveries related to Dystroglycanopathy.

We are honored to support groundbreaking discoveries related to LAMA2-Related Dystrophy.

The MAGIC research team welcomes iMM to the consortium to add a cellular perspective to muscle-on-chip technologies

Long-awaited publication about the Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy now published.

We are honored to support groundbreaking discoveries related to SELENON-Related Disorders.

Explore an in-depth interview with Gustavo Dziewczapolski, Doctor in Neuropharmacology and Scientific Director of Cure CMD. Gain insights in

Cure CMD’s Scientific Director, Dr. Gustavo Dziewczapolski, provides a recap of his attendance at the 8th International Myology Congress in

We are honored to support groundbreaking discoveries in Collagen 6-Related Dystrophies (COL6-RD).

Expanding on nearly a decade of partnership in the life sciences, the Translational Sciences team at UPMC Enterprises and the University...