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pro vyhledávání: '"Hannah Y. Coletti"'
Publikováno v:
Orphanet Journal of Rare Diseases
Background Newborn screening for mucopolysaccharidosis type I (MPS I) shows promise to improve outcomes by facilitating early diagnosis and treatment. However, diagnostic tests for MPS I are of limited value in predicting whether a child will develop
Autor:
Maria L. Escolar, Michele D. Poe, Hannah Y. Coletti, Mieke Aldenhoven, Joanne Kurtzberg, Karina Yelin
Publikováno v:
JIMD Reports ISBN: 9783662466995
Hurler syndrome is characterized by progressive multisystem deterioration leading to early death in childhood. This prospective study evaluated the long-term outcomes of patients with Hurler syndrome who underwent umbilical cord blood transplantation
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2dcd8a4cbb506b499275437995cfe238
https://europepmc.org/articles/PMC4375128/
https://europepmc.org/articles/PMC4375128/