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.