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Cleveland Clinic Tall Rounds: Post‐diagnostic Monitoring of Cardiac Amyloidosis
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Cleveland Clinic Tall Rounds: Treating Confirmed ATTR
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Addressing DMD Time to Diagnosis: Microlearning for Pediatricians & Primary Care
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Hemophilia Gene Therapy: Shared Decision Making - Clinical Companion Guide
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Risks and Benefits of Hemophilia Gene Therapy: An EXPLORE Learning Experience
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Conference Updates in DMD and LGMD (WMS and MDA)
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Limb-Girdle Muscular Dystrophy EXPLORE Module
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Rett Syndrome: Delayed Diagnosis, Patient Perspectives & Evolving Treatment - Rapid Recap
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Rett Syndrome: Delayed Diagnosis, Patient Perspectives & Evolving Treatment - Slide Deck
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Rett Syndrome: Delayed Diagnosis, Patient Perspectives & Evolving Treatment
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Contemporary Strategies: Diagnosing ATTR Amyloidosis
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Emerging Biomarker Strategies for Lysosomal Storage Diseases
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A Focus on Fabry Disease: An EXPLORE™ eLearning Module
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Understanding Lysosomal Storage Disorders: From Newborn Screening to Treatments
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DMD and Gene Therapy: What's on the Horizon?
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Updates in SMA: Cure SMA Conference Coverage (CME/CE)
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Conference Coverage on DMD and LGMD: Latest Developments
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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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Updates in SMA: WMS Conference Coverage (CME/CE)
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Updates in SMA: WMS Conference Coverage
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SMA: Introduction and Key Concepts
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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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Updates in SMA from Charlotte, North Carolina
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Gene Therapy for Treatment of Hemophilia: Other Strategies and Targets
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Technology Improving SMA Patients’ Quality of Life
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Nusinersen Treatment Outcomes in Later-Onset SMA
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Patients with Severe SMA Need Additional Respiratory Management
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Clear Expectations Needed in SMA Patient Management
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Improvements in Quality of Life for Patients with Neuromuscular Diseases
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Updates in SMA and Gene Therapy from Copenhagen, Denmark
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The Changing Landscape of SMA–Updates from Copenhagen, Denmark
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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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The Changing Landscape of SMA–Updates from Anaheim, CA
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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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A 30 y/o Female w/2-year History of Shoulder Weakness and Elevated AST and ALT
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32 y/o Male w/Progressive Weakness, Dilated Cardiomyopathy & Respiratory...
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A 60 y/o Male w/4-year History Of Shoulder Weakness
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Approach to Genetic Testing in Myopathies
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Molecular Diagnosis of Limb-Girdle Muscle Weakness

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