AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease
NEJM 392:2438-2446, 2477, Ma,X.,et al, 2025
Lentiviral Gene Therapy for Cerebral adrenoleukodystrophy
NEJM 391:1302-1312,1358, Eichler,F.,et al, 2024
Muscular Dystrophies
Lancet 394:2025-2038, Mercuri, E.,et al, 2019
Clinical Imaging Factors Associated with Infarct Progression in Patients with Ischemic Stroke During Transfer for Mechanical Thrombectomy
JAMA Neurol 74:1361-1367,1298, Boulouis, G.,et al, 2017
Progressive Multifocal Leukoencephalopathy and Relapsing-Remitting Multiple Sclerosis: A Comparative Study
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