Gene therapy in rare diseases: the benefits and challenges...

Orphanet Journal of Rare Diseases 2018 / 12 Vol. 13; Iss. 1

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

Stirnadel-Farrant, Heide, Kudari, Mahesh, Garman, Nadia, Imrie, Jessica, Chopra, Bikramjit, Giannelli, Stefania, Gabaldo, Michela, Corti, Ambra, Zancan, Stefano, Aiuti, Alessandro, Cicalese, Maria Pia
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Volume:
13
Language:
english
Journal:
Orphanet Journal of Rare Diseases
DOI:
10.1186/s13023-018-0791-9
Date:
December, 2018
File:
PDF, 669 KB
english, 2018
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