November 9, 2020 Results from all cohorts of PRECISION-HD1 and PRECISION-HD2 clinical trials and initial OLE data on track for 1Q 2021Dosing in three new clinical trials with novel compounds […]
11/05/20 4:02 PM EST — Dosing with SRP-9001 commercial-process material to proceed following discussions with U.S. FDA — — Clinical dosing is expected to begin before the end of 2020 — CAMBRIDGE, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in […]
Learn about PTC Cares™, a support program dedicated to helping patients, caregivers, and prescribers understand the prescription process, financial assistance, and insurance information for PTC products. You’ll be prompted to […]
-Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants- -Solid receives $40 million upfront via equity investment at a premium; up […]
The CureDuchenne Time Capsule Video is a beautiful compilation of interviews conducted at the 2019 FUTURES Conference in Anaheim, CA. We asked families to share their stories with us, as […]
Dear Duchenne community, Since 2016, Santhera has been conducting one of the largest clinical trials in Duchenne muscular dystrophy – the SIDEROS study. SIDEROS is a phase III double-blind, randomized, […]
La 2da parte incluirá los siguientes temas: Educación para el cuidador Sugerencias para los cuidadores – mejores prácticas físicas, mentales y emocionales Anécdota familia & construcción comunitaria Se le pedirá […]
La 1ra parte incluira los siguientes temas: Acceso al Cuidado Multidisciplinario Guia nutricional para Individuos con Duchenne Programa de Asistencia Familiar Duchenne (Duchenne Family Assistance Program) Se le pedirá que […]
This month’s Catabasis Connection:
