2023年MDA Kickstart计划获得了先天性肌无力综合征的罕见儿科疾病和孤儿药称号。 2023 MDA Kickstart program receives Rare Pediatric Disease & Orphan Drug designations for Congenital Myasthenic Syndrome.

肌肉萎缩症协会的 MDA Kickstart 计划已获得 FDA 的罕见儿科疾病和由 CHAT 基因缺陷引起的先天性肌无力综合征孤儿药的指定。 The Muscular Dystrophy Association's MDA Kickstart program has received FDA designations for Rare Pediatric Disease and Orphan Drug for Congenital Myasthenic Syndrome caused by CHAT gene deficiency. 该方案于2023年启动,旨在加速发展影响美国不到1 000人的超危险神经肌肉疾病的基因疗法。 Launched in 2023, the program aims to expedite development of gene therapies for ultra-rare neuromuscular diseases affecting fewer than 1,000 people in the U.S. 该倡议与Forge生物学组织合作,支持制造业。 The initiative collaborates with Forge Biologics for manufacturing support. 优先审查券方案将于2024年12月20日到期,除非国会重新授权。 The Priority Review Voucher program is set to expire on December 20, 2024, unless reauthorized by Congress.