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4. INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy. Dowling JJ; Müller-Felber W; Smith BK; Bönnemann CG; Kuntz NL; Muntoni F; Servais L; Alfano LN; Beggs AH; Bilder DA; Blaschek A; Duong T; Graham RJ; Jain M; Lawlor MW; Lee J; Coats J; Lilien C; Lowes LP; MacBean V; Neuhaus S; Noursalehi M; Pitts T; Finlay C; Christensen S; Rafferty G; Seferian AM; Tsuchiya E; James ES; Miller W; Sepulveda B; Vila MC; Prasad S; Rico S; Shieh PB; J Neuromuscul Dis; 2022; 9(4):503-516. PubMed ID: 35694931 [TBL] [Abstract][Full Text] [Related]
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