Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
NEJM 392:1609-1620, Fumagalli,F.,et al, 2025
Risdiplam in Presymptomatic Spinal Muscular Atrophy
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Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy
NEJM 391:1256-1258, Laugwitz,L.,et al, 2024
Distinctive Imaging in a Paucisymptomatic Child with Leukodystrophy
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Hemorrhagic Transformation of Childhood Arterial Ischemic Stroke
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"Im fine; Im just waiting for my disease" The New and Growing Class of Presymptomatic Patients
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Brain Abnormalities in Neuromyelitis Optica
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The Tuberous Sclerosis Complex
NEJM 355:1345-1356, Crino,P.B.,et al, 2006
Silent Infarcts in Children With Sickle Cell Anemia and Abnormal Cerebral Artery Velocity
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The Spectrum of Brain MR Abnormalities in Sickle-Cell Disease:A Report from the Cooperative Study of Sickle Cell Disease
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