by Madaline Spencer | Oct 25, 2024
Cathy Turner, Project Manager for DMD Care UK at the John Walton Muscular Dystrophy Research Center at Newcastle University, discusses the TREAT-NMD Advisory Committee for Therapeutics (TACT). TACT was established in 2009 with the goal of providing...
by Madaline Spencer | Oct 24, 2024
Charlotte Handberg, PhD, Senior Researcher at the National Rehabilitation Center for Neuromuscular Diseases in Denmark, and Associate Professor at Aarhus University, discusses results from a survey of parents’ hopes, worries, and needs for their children with spinal...
by Madaline Spencer | Oct 22, 2024
Emma Robinson, Neuromuscular Research Physiotherapist at the John Walton Muscular Dystrophy Research Center at Newcastle University, discusses how losing the ability to stand affects dysferlinopathy patients’ social participation. Dysferlin-related...
by Madaline Spencer | Oct 17, 2024
Cathy Turner, Project Manager for DMD Care UK at the John Walton Muscular Dystrophy Research Center at Newcastle University, discusses the Duchenne Education for Care and Research Initiative (DECRI). DMD is a rare genetic disorder due to mutations in the...
by Madaline Spencer | Oct 10, 2024
David Weber, MD, Attending Physician and Medical Director of the Center for Bone Health at Children’s Hospital of Philadelphia, discusses investigational treatment INZ-701 for ENPP1 deficiency. Correction: Dr. Weber cited the genetic incidence of ENPP1...