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AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease
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Unmasking Cerebrotendinous Xanthomatosis, Clinical Recognition of a Treatable Cause of Progressive Ataxia
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New Treatments for Migraine: CGRP Monoclonal Antibodies, Gepants, and Ditans
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Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia
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Comparative Effects of Drug Interventions for the Acute Management of Migraine Episodes in Adults:Systematic Review and Network Meta-Analysis
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Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy
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Lentiviral Gene Therapy for Cerebral adrenoleukodystrophy
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A 65-Year-Old Woman with Isolated Macroglossia as the Initial Presentation of a Rare Disease
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A Young Adult Man with Cognitive Changes, Gait Difficulty, and Renal Insufficiency
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Neuroimaging Features of Biotinidase Deficiency
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Neonatal Seizures
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Adult Patient Presenting with Spine Pain Following a Motor Vehicle Accident
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Atogepant for the Preventive Treatment of Chronic Migraine (PROGRESS): A Randomised, Double-blind, Placebo-controlled, Phase 3 Trial
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Current and Emerging Issues in Wilsons Disease
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Hereditary Hemorrhagic Telangiectasia, Clinical Presentations, and Management
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Lentiviral Haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
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A 6-Year-Old Girl with Progressive Toe Walking
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Brain Tumors in Children
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A 23-Year-Old Woman Presenting with Cognitive Impairment and Gait Disturbance
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Updates on Sturge-Weber Syndrome
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Acute Treatment for Migraine
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Atogepant for the Preventive Treatment of Migraine
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Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
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A 22-Year-Old Man with Progressive Bilateral Visual Loss
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One-Year Sustained Efficacy of Erenumab in Episodic Migraine
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Muscle Stiffness, Gait Instability, and Liver Cirrhosis in Wilsons Disease
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Fibromuscular Dysplasia and Its Neurologic Manifestations
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A Teenager with Persistent Headache
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Remigepant, an Oral Calcitonin Gene-Related Peptide Receptor Antagonist, for Migraine
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Trial of Galcanezumab in Prevention of Episodic Cluster Headache
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Pes Cavus and Neuropathy
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Ehlers-Danlos Syndromes
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Targeting Calcitonin Gene-Related Peptide: A New Era in Migraine Therapy
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