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Results 1 - 43 of 43
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The Delicate Balance of Brain Cholesterol - Online Module
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The Delicate Balance of Brain Cholesterol - Clinical Companion Guide
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The Delicate Balance of Brain Cholesterol - Slide Deck
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The Delicate Balance of Brain Cholesterol - Rapid Recap
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The Delicate Balance of Brain Cholesterol - Live Meeting Recording
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Limb-Girdle Muscular Dystrophy EXPLORE Module
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Emerging Biomarker Strategies for Lysosomal Storage Diseases
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Understanding Lysosomal Storage Disorders: From Newborn Screening to Treatments
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Updates in Duchenne Muscular Dystrophy: A Focus on Emerging Gene Therapy
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LGMD: Mutation-specific Nomenclature
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LGMD Symptomatology
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LGMD Mutations and Disease Types
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Tests and Outcomes - DMD
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Genes, Exons, and the Pathophysiology of DMD
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The Natural History of DMD
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Key Considerations: Advances in Gene Therapy for Hemophilia
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Integrating Emerging Treatments for Hemophilia A
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The Safety of Hemophilia Gene Therapy Now and in the Future
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Viral Vectors Gene Therapy Podcasts
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WFH Gene Therapy Registry
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HTC Readiness: A Multidisciplinary Perspective
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HTC Readiness: A Global Perspective
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Gene Therapy: Patient Support, Patient Counseling, and Monitoring
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Gene Therapy: Update on Efficacy of Clinical Trials
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WMS Conference Coverage on DMD and LGMD: Experts Reporting From the Field
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Conference Coverage on DMD & LGMD
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Hereditary Transthyretin Amyloidosis: Case Studies
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Understanding Lysosomal Storage Disorders
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hATTR Amyloidosis: You and Your Family
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Gene Therapy for Treatment of Hemophilia: AAV Vector Gene Therapy Application to Hemophilia
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Gene Therapy for Treatment of Hemophilia: An Introduction to AAV Vector Gene Transfer
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SMA Updates from Around the World
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Gene Therapy for Treatment of Hemophilia: Other Strategies and Targets
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Gene Therapy for Treatment of Hemophilia: Common Concerns in Gene Therapy
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Are You Ready for Gene Replacement Therapy?
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Gene Therapy for SMA: An Emerging Treatment Option for a Devastating Disease
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SMA: Data, Insights and Practice
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From Bench to Bedside: Gene Therapy Approaches for Rare Diseases
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What’s New in Spinal Muscular Atrophy? Reporting from Dallas
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SMA Updates from Los Angeles
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Updates in SMA and Gene Therapy from Arlington, VA
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Genetics and Personalized Medicine for Patients with Pulmonary Fibrosis
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Biomarkers for IPF

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