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...
by Madaline Spencer | Oct 3, 2024
Kurt Gunter, MD, Senior Vice President and Chief Medical Officer at Inozyme Pharma, discusses data presented on ENPP1 deficiency and ABCC6 deficiency at the 2024 American Society for Bone and Mineral Research meeting. ENPP1 ENPP1 deficiency is a rare...
by Madaline Spencer | Oct 1, 2024
Paul Bolno, CEO, and Anne-Marie Li-Kwai-Cheung, Chief Development Officer, of Wave Life Sciences, discuss interim data from the ongoing FORWARD-53 clinical trial for Duchenne muscular dystrophy (DMD). DMD affects the muscles, leading to progressive...
