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Proceedings of the National Academy of Sciences of the United States of America|January 30, 2013
DNA and virus particle vaccination protects against acquisition and confers control of viremia upon heterologous simian immunodeficiency virus challengeVainav Patel, Rashmi Jalah, Viraj Kulkarni, et al.Neuromuscular Disorders : NMD|January 6, 2024
270th ENMC International Workshop: Consensus for SMN2 genetic analysis in SMA patients 10-12 March, 2023, Hoofddorp, the NetherlandsEmanuela Abiusi, Mar Costa-Roger, Enrico Silvio Bertini, et al.The Lancet. Neurology|March 20, 2024
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trialEugenio Mercuri, Juan J Vilchez, Odile Boespflug-Tanguy, et al.Therapeutic Advances in Rare Disease|September 22, 2025
Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO studyPerry B Shieh, Wendy Hughes, Marie Wood, et al.Microorganisms|December 2, 2020
Anti-SARS-CoV-2 Antibody Responses in Convalescent Plasma Donors Are Increased in Hospitalized Patients; Subanalyses of a Phase 2 Clinical StudyEvangelos Terpos, Marianna Politou, Theodoros N Sergentanis, et al.American Journal of Human Genetics|October 24, 2003
Mutations in a gene encoding a novel SH3/TPR domain protein cause autosomal recessive Charcot-Marie-Tooth type 4C neuropathyJan Senderek, Carsten Bergmann, Claudia Stendel, et al.The Lancet. Child & Adolescent Health|November 10, 2021
Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort studyClaudia Weiß, Andreas Ziegler, Lena-Luise Becker, et al.The American Journal of Psychiatry|October 29, 2021
Stanford Neuromodulation Therapy (SNT): A Double-Blind Randomized Controlled TrialEleanor J Cole, Angela L Phillips, Brandon S Bentzley, et al.Journal of Neuromuscular Diseases|November 21, 2019
Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1Astrid Pechmann, Matthias Baumann, Günther Bernert, et al.Physical Review Letters|October 1, 2013
Generation of coherent 19- and 38-nm radiation at a free-electron laser directly seeded at 38 nmS Ackermann, A Azima, S Bajt, et al.Pageof 102