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Current and Emerging Issues in Wilsons Disease
NEJM 389:922-938, Roberts,E.A. & Schilsky, M.L., 2023
Muscular Dystrophies
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Spinal Muscular Atrophy A Timely Review
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Diagnosis and New Treatments in Muscular Dystrophies
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Diagnosis and Therapy in Neuromuscular Disorders: Diagnosis and New Treatments in Mitochondrial Diseases
JNNP 80:943-953, Rahman,S. &Hanna,M.G., 2009
Gene Therapy for Duchenne Muscular Dystrophy: Expectations and Challenges
Arch Neurol 64:1236-1241, Rodino-Klapac,L.R.,et al, 2007
Glycogen-Storage Disease Type II
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Gene Transfer for Neurologic Disease
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Neurovirological Methods and Their Applications
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Neurogene Therapy for the 21st Century
Arch Neurol 57:54, Brady,R.O., 2000
X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy
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Gene Therapy for Cerebral Vascular Disease
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Gene Therapy for Neurologic Disease
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