Browsing by Author Mavilio, F

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Issue DateTitleAuthor(s)
15-Nov-2023Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trialShieh, PB; Kuntz, NL; Dowling, JJ; Müller-Felber, W; Bönnemann, CG; Seferian, AM; Servais, L; Smith, BK; Muntoni, F; Blaschek, A; Foley, AR; Saade, DN; Neuhaus, S; Alfano, LN; Beggs, AH; Buj-Bello, A; Childers, MK; Duong, T; Graham, RJ; Jain, M; Coats, J; MacBean, V; James, ES; Lee, J; Mavilio, F; Miller, W; Varfaj, F; Murtagh, M; Han, C; Noursalehi, M; Lawlor, MW; Prasad, S; Rico, S