Browsing by Subject X-linked myotubular myopathy
Showing results 1 to 2 of 2
Issue Date | Title | Author(s) |
---|---|---|
2024 | An Algorithm for Discontinuing Mechanical Ventilation in Boys with X-Linked Myotubular Myopathy After Positive Response to Gene Therapy: The ASPIRO Experience | Graham, RJ; Amin, R; Demirel, N; Edel, L; Lilien, C; MacBean, V; Rafferty, GF; Sawnani, H; Schon, C; Smith, BK; Syed, F; Sarazen, M; Prasad, S; Rico, S; Perez, GF |
7-Jun-2022 | 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 |