Resources


Understanding New Discoveries in Genomic Medicine

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Genomic Medicine: Unique Considerations for Rare Disease Clinical Trials

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Genomic Medicine: Understanding Genome Engineering, A Focus on Genome Regulation

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Genomic Medicine: Understanding Genome Engineering, A Focus on Genome Editing

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Genomic Medicine: Understanding Genome Engineered Cell Therapies

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American Association of Kidney Patients (AAKP)

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Centers for Disease Control and Prevention

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Cooleys Anemia Foundation

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Fabry International Network

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Fabry Support & Information Group (FSIG)

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Hemophilia Federation of America

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National Fabry Disease Foundation

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National Hemophilia Foundation

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National Organizations for Rare Disorders (NORD)

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Thalassaemia International Federation (TIF)

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The Society for Mucopolysaccharide Diseases (MPS Society)

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World Federation of Hemophilia

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