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 therapies.

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

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, who are united...