サイトウ　カヨコ   Saito Kayoko   齋藤　加代子    所属   医学部 医学科（東京女子医科大学病院）    職種   特任教授
論文種別	原著
言語種別	英語
査読の有無	査読あり
表題	Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.
掲載誌名	正式名：Annals of clinical and translational neurology 略　 称：Ann Clin Transl Neurol ISSNコード：23289503/23289503
掲載区分	国外
巻・号・頁	10(11),pp.2155-2160
国際共著	国際共著
著者・共著者	Proud Crystal M, Mercuri Eugenio, Finkel Richard S, Kirschner Janbernd, De Vivo Darryl C, Muntoni Francesco, Saito Kayoko, Tizzano Eduardo F, Desguerre Isabelle, Quijano-Roy Susana, Benguerba Kamal, Raju Dheeraj, Faulkner Eric, Servais Laurent
発行年月	2023/09
概要	We sought to devise a rational, systematic approach for defining/grouping survival motor neuron-targeted disease-modifying treatment (DMT) scenarios. The proposed classification is primarily based on a two-part differentiation: initial DMT, and persistence/discontinuation of subsequent DMT(s). Treatment categories were identified: monotherapy add-on, transient add-on, combination with onasemnogene abeparvovec, bridging to onasemnogene abeparvovec, and switching to onasemnogene abeparvovec. We validated this approach by applying the classification to the 443 patients currently in the RESTORE registry and explored the demographics of these different groups of patients. This work forms the basis to explore the safety and efficacy profile of the different combinations of DMT in SMA.
DOI	10.1002/acn3.51889
PMID	37691296