Hematologic & Lysosomal Storage Disorders Resource Library
Explore examples of research, developmental milestones, and investigational treatment approaches targeting the liver.
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Hemophilia A
HemophiliaLearn how the frequency of bleeding episodes in patients with hemophilia A determines the severity of disease. See how it is diagnosed and managed.
Pompe Disease
PompeReview clinical characteristics, diagnosis and testing options, management, and more for Pompe disease.
AAV Gene Therapy Research Animation
Gene TherapyAdeno-associated viral (AAV) vectors are designed to encapsulate genetic material for the potential treatment of genetic diseases. Explore the science behind AAV gene therapy research.
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Gene Therapy Strategies
Gene TherapyLearn how genetic therapy approaches can potentially correct genetic mutations to provide functional protein.
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Gene Therapy and the Immune Response
Gene TherapyAdministering AAV vectors can activate different branches of the immune system. Learn about this process and research to develop immunosuppressive techniques.
Supplemental Resources
Explore additional hematologic and lysosomal storage disorder resources to supplement conversations with your patients.
Annual Hematology and Lysosomal Storage Disorders Events
At Spark Therapeutics, we’re striving to address unmet needs in rare genetic diseases. Here are some events relevant to our research.
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Get information about Spark products and therapeutic areas or request a discussion with a medical science liaison.
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