Salvador Rico

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

Perry B. Shieh, Nancy L. Kuntz, Wolfgang Müller‐Felber et al. · The Lancet Neurology · 2023 · 120 citations · Full text

Medicine, Molecular Medicine, Pharmacotherapy +15

Red blood cell concentrates treated with the amustaline (S‐303) pathogen reduction system and stored for 35 days retain post‐transfusion viability: results of a two‐centre study

José A. Cancelas, Jerome L. Gottschall, Neeta Rugg et al. · Vox Sanguinis · 2017 · 45 citations · Full text

Medicine, Hematology, Cell Therapy +16

Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis

Robert J. Graham, Francesco Muntoni, Imelda Hughes et al. · Archives of Disease in Childhood · 2019 · 43 citations · Full text

Medicine, Pediatrics, Clinical Epidemiology +16

Red blood cells treated with the amustaline (S‐303) pathogen reduction system: a transfusion study in cardiac surgery

V. Brixner, Katharina Madlener, Markus Müller et al. · Transfusion · 2018 · 40 citations · Full text

Medicine, Hematology, Surgery +17

DRAVET ENGAGE. Parent caregivers of children with Dravet syndrome: Perspectives, needs, and opportunities for clinical research

Clara Juandó‐Prats, Emma S. James, Deborah A. Bilder et al. · Epilepsy & Behavior · 2021 · 36 citations · Full text

Nursing, Child Health, Pediatrics +17