Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry...
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID
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Publisher
England: BioMed Central Ltd
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Language
English
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Publisher
England: BioMed Central Ltd
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Contents
Strimvelis (autologous CD34+ cells transduced to express adenosine deaminase [ADA]) is the first ex vivo stem cell gene therapy approved by the European Medicines Agency (EMA), indicated as a single treatment for patients with ADA-severe combined immunodeficiency (ADA-SCID) who lack a suitable matched related bone marrow donor. Existing primary imm...
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Full title
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID
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TN_cdi_doaj_primary_oai_doaj_org_article_a8893f1115da49d982593a6f5bd7315f
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_a8893f1115da49d982593a6f5bd7315f
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-018-0791-9
