Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial. - Lawlor MW; Schoser B; Margeta M; Sewry CA; Jones KA; Shieh PB; Kuntz NL; Smith BK; Dowling JJ; Müller-Felber W; Bönnemann CG; Seferian AM; et al. (EBioMedicine, 2024)