Accelerated Approval pathway for our ST-920 gene therapy product candidate for the treatment of Fabry disease.
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Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
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Overview
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10 Things to Know About Clinical Trials
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Education
A Closer Look at Gene Therapy
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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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Fabry International Network
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Fabry Support & Information Group (FSIG)
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Global Genes
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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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World Federation of Hemophilia
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