Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID
出版年份 2018 全文链接
标题
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID
作者
关键词
Adenosine deaminase deficiency, Severe combined immunodeficiency, Gene therapy, Haematopoietic stem cell transplantation, Transplantation, Autologous, Pharmacovigilance
出版物
Orphanet Journal of Rare Diseases
Volume 13, Issue 1, Pages -
出版商
Springer Nature
发表日期
2018-04-19
DOI
10.1186/s13023-018-0791-9
参考文献
相关参考文献
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