The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Resolaris as a potential treatment for…
Articles by Margarida Azevedo, MSc
A researcher at the University of Texas Southwestern Medical Center has been awarded a $250,000 grant to study CRISPR/Cas9…
Yale New Haven Children’s Hospital’s (YNHCH) Muscular Dystrophy Program has been named a Certified Duchenne Care Center by Parent…
The New Jersey Institute of Technology (NJIT) and Talem Technologies will explore the potential of robotic technology to assist…
The European Commission has granted Orphan Drug Designation to Benitec Biopharma’s BB-301, an investigational treatment for oculopharyngeal muscular dystrophy…
CureDuchenne Cares, a community-based program providing educational and outreach workshops for parents and others caring for people with Duchenne muscular…
Many patients with myotonic dystrophy type 1 (DM1) have sleep and respiratory disorders and may benefit from targeted therapy. New…
The European Commission (EC) has decided to renew the conditional marketing authorization for Translarna (ataluren) to treat certain nonsense mutation Duchenne muscular…
Researchers found notable differences in the access to appropriate care for Duchenne’s muscular dystrophy (DMD) patients in seven European countries. In a…
Children with special healthcare needs, such as those with muscular dystrophy (MD), benefit from at-home care, but at high…