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Metabolic Lipid Muscle Disorders: Biomarkers and Treatment
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Niemann-Pick Disease Type C from Bench to Bedside
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Chronic Cardiomyopathy and WEakness or Acute Coma in Children with a Defect in Carnitine Uptake
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Carnitine Deficiency Acute Postpartum Crisis
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Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
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Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy
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Adult Patient Presenting with Spine Pain Following a Motor Vehicle Accident
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A 23-Year-Old Woman Presenting with Cognitive Impairment and Gait Disturbance
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Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
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Clinicopathologic Conference, Statin-Associated Autoimmune Myopathy
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Pes Cavus and Neuropathy
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Long-Term Benefit of Enzyme Replacement Therapy in Pompe Disease
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Statin-Associated Autoimmune Myopathy
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A 38-Year-Old Man with Respiratory Failure and Progressive Leg Weakness
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Small Fiber Neuropathy in Fabry Disease:A Review of Pathophysiology and Treatment
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Anti-HMGCR Autoantibodies in European Patients with Autoimmune Necrotizing Myopathies
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The Spectrum of Statin Myopathy
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"Im fine; Im just waiting for my disease" The New and Growing Class of Presymptomatic Patients
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Statin Induced Myopathy
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Progressive Myoclonic Epilepsies: A Review of Genetic and Therapeutic Aspects
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Insights Into the Diagnosis and Treatment of Lysosomal Storage Diseases
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AAC/AHA/NHLBI Clinical Advisory on the Use and Safety of Statins
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Enzyme Replacement Therapy in Fabry Disease
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Follow-up of Nine Patients with Hurler Syndrome After Bone Marrow Transplantation
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Fucosidosis Revisited:A Review of 77 Patients
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Bone-Marrow Transplantation for Neurovisceral Storage Disorders
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Enzyme Replacement in Nervous Tissue After Allogeneic Bone-Marrow Transplantation for Fucosidoisis in Dogs
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McArdle's Disease in the 1980s
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Diagnosis and Management of ANCA-Associated Vasculitis
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Expanding Clinical Spectrum an Anti-GQ1b Antibody Syndrome, A Review
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Clinicopathologic Conference, Infective Endocarditis Due to Haemophilus Parainfluenza
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Progressive Camptocormia with Head Drop and Dysphagia
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Clinicopathologic Conference, Hypocalcemic Myopathy Due to Hypoparathyroidism
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Extrapulmonary Manifestations of Sarcoidosis
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Stroke Prevention in Symptomatic Large Artery Intracranial Atherosclerosis Practice Advisory
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Epidemiology, Survival, and Clinical Characteristics of Inclusion Body Myositis
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Clinicopathologic Conference, Granulomatosis with Polyangiitis
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Spinal Muscular Atrophy
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A Comparison of Two LDL Cholesterol Targets after Ischemic Stroke
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Severe Neurological Toxicity of Immune Checkpoint Inhibitors: Growing Spectrum
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When the Temporal Artery Biopsy is Negative
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Severe Neurological Toxicity of Immune Checkpoint Inhibitors: Growing Spectrum
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Covd-19-Associated Myopathy Caused by Type 1 Interferonopathy
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Cholesterol Lowering and Prevention of Stroke
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Neuromuscular Adverse Events Associated with Anti-PD-1 Monoclonal Antibodies
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