Natural history, outcome measures and trial readiness in LAMA2-related muscular dystrophy and SELENON-related myopathy in children and adults: protocol of the LAST STRONG study
LAMA2相关性肌营养不良症和SELENON相关性肌病在儿童和成人中的自然病程、结局指标和试验准备情况:LAST STRONG研究方案
期刊:BMC Neurology
影响因子:2.2
doi:10.1186/s12883-021-02336-z
Bouman, Karlijn; Groothuis, Jan T; Doorduin, Jonne; van Alfen, Nens; Udink Ten Cate, Floris E A; van den Heuvel, Frederik M A; Nijveldt, Robin; van Tilburg, Willem C M; Buckens, Stan C F M; Dittrich, Anne T M; Draaisma, Jos M T; Janssen, Mirian C H; Kamsteeg, Erik-Jan; van Kleef, Esmee S B; Koene, Saskia; Smeitink, Jan A M; Küsters, Benno; van Tienen, Florence H J; Smeets, Hubert J M; van Engelen, Baziel G M; Erasmus, Corrie E; Voermans, Nicol C