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 15, 2024
Stefanie Mason, MD, Senior Medical Director and Clinical Development Lead for Duchenne muscular dystrophy (DMD) at Sarepta Therapeutics, discusses results from the phase 3 EMBARK clinical trial for DMD. DMD is a rare genetic disorder due to mutations in...
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...
by Madaline Spencer | Oct 9, 2024
Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, at Alexion, discusses positive data for an investigational treatment for ATTR with cardiomyopathy (ATTR-CM). AATR-CM is a rare amyloidosis caused by aging or genetic...
