The Efficacy and Safety of Different Targeted Drugs for the Treatment of Generalized Myasthenia Gravis: A Systematic Review and Bayesian Network Meta-analysis
Authors (first, second and last of 6)
Collection
Rare Diseases
A collection of articles discussing the pharmacological treatment of rare diseases of relevance to neurologists and psychiatrists.
Articles (22 in this collection)
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Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future
Authors (first, second and last of 7)
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The Safety of Deutetrabenazine for Chorea in Huntington Disease: An Open-Label Extension Study
Authors (first, second and last of 14)
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Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model
Authors (first, second and last of 10)
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Efficacy, Retention and Tolerability of Everolimus in Patients with Tuberous Sclerosis Complex: A Survey-Based Study on Patients’ Perspectives
Authors (first, second and last of 38)
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Pathophysiology and Treatment of Non-motor Dysfunction in Amyotrophic Lateral Sclerosis
Authors (first, second and last of 8)
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Expanding the Treatment Landscape for Lennox-Gastaut Syndrome: Current and Future Strategies
Authors
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Adjunctive Cannabidiol in Patients with Dravet Syndrome: A Systematic Review and Meta-Analysis of Efficacy and Safety
Authors (first, second and last of 7)
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An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials
Authors (first, second and last of 9)
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Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1
Authors (first, second and last of 6)
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Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses
Authors (first, second and last of 4)
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Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management
Authors (first, second and last of 8)
