Current and Emerging Issues in Wilsons Disease
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X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy
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Gene Therapy for Cerebral Vascular Disease
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Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
NEJM 392:1609-1620, Fumagalli,F.,et al, 2025
Parkinsons Disease
NEJM 391:442-452, Tanner,C.M. & Ostrem,J.L., 2024
Lentiviral Gene Therapy for Cerebral adrenoleukodystrophy
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Muscular Dystrophies
Lancet 394:2025-2038, Mercuri, E.,et al, 2019
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017
Spinal Muscular Atrophy A Timely Review
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Diagnosis and New Treatments in Muscular Dystrophies
JNNP 80:706-714, Manzur,A.Y. &Muntoni,F., 2009
Diagnosis and Therapy in Neuromuscular Disorders: Diagnosis and New Treatments in Mitochondrial Diseases
JNNP 80:943-953, Rahman,S. &Hanna,M.G., 2009
Gene Transfer for Neurologic Disease
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Neurovirological Methods and Their Applications
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