Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

BACKGROUND: 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 su...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Stirnadel-Farrant, Heide, Kudari, Mahesh, Garman, Nadia, Imrie, Jessica, Chopra, Bikramjit, Giannelli, Stefania, Gabaldo, Michela, Corti, Ambra, Zancan, Stefano, Aiuti, Alessandro, Cicalese, Maria Pia, Batta, Rohit, Appleby, Jonathan, Davinelli, Mario, Ng, Pauline
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5889583/
https://ncbi.nlm.nih.gov/pubmed/29625577
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0791-9
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