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Autor:
Simona D'Amore, Kathleen Page, Aimée Donald, Khadijeh Taiyari, Brian Tom, Patrick Brian Deegan, Chong Yew Tan, Kenneth Poole, Simon Allan Jones, Atul Mehta, Derralynn Hughes, Reena Sharma, Robin Henry Lachmann, Aunpam Chakrapani, Tarekegn Geberhiwot, Saikat Santra, Timothy Martin Cox
Background The Gaucher Investigative Therapy Evaluation (GAUCHERITE) is a national clinical cohort of 250 patients aged 5–87 years with Gaucher disease - an ultra-rare genetic disorder. To inform clinical decision-making and improve pathophysiologi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::f50a04e14b992758eb52e22b6b7f140c
https://doi.org/10.21203/rs.3.rs-543669/v1
https://doi.org/10.21203/rs.3.rs-543669/v1