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European Stroke Journal|July 31, 2026
Primary versus elective percutaneous transluminal angioplasty with stenting for symptomatic intracranial atherosclerotic stenosis: a 18-centre retrospective cohort study using the pEGASUS-HPC stentAli Khanafer, Ahmed Ayad, Mohammad Almohammad, et al.
Journal of Neuromuscular Diseases|June 13, 2022
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular MyopathyJames J Dowling, Wolfgang Müller-Felber, Barbara K Smith, et al.
Journal of Neuromuscular Diseases|November 24, 2025
Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidenceClaudia Weiß, Katharina Vill, Matthias Baumann, et al.
Journal of Child Neurology|November 17, 2010
Consensus statement on standard of care for congenital muscular dystrophiesChing H Wang, Carsten G Bonnemann, Anne Rutkowski, et al.
Nature Medicine|May 23, 2018
HIV vaccine candidate activation of hypoxia and the inflammasome in CD14+ monocytes is associated with a decreased risk of SIVmac251 acquisitionMonica Vaccari, Slim Fourati, Shari N Gordon, et al.
Blood|September 22, 2025
Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency: an EBMT-IEWP retrospective studyUli S Herrmann, Matthias Felber, Austen Worth, et al.
Plos Pathogens|July 6, 2021
Interleukin-15 response signature predicts RhCMV/SIV vaccine efficacyFredrik Barrenäs, Scott G Hansen, Lynn Law, et al.
Blood|July 3, 2020
Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adultsRobert Chiesa, Junfeng Wang, Henric-Jan Blok, et al.
JAMA Pediatrics|April 8, 2024
Clinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled TrialOliver Schwartz, Katharina Vill, Michelle Pfaffenlehner, et al.
Ebiomedicine|December 12, 2023
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 trialMichael W Lawlor, Benedikt Schoser, Marta Margeta, et al.
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