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Improvement in time to treatment, but not time to diagnosis, in patients with mucopolysaccharidosis type I

OBJECTIVE: Early diagnosis and treatment initiation are important factors for successful treatment of mucopolysaccharidosis type I (MPS I). The purpose of this observational study was to assess whether age at diagnosis and time to first treatment for individuals with MPS I have improved over the las...

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Pubblicato in:Arch Dis Child
Autori principali: Giugliani, Roberto, Muschol, Nicole, Keenan, Hillary A., Dant, Mark, Muenzer, Joseph
Natura: Artigo
Lingua:Inglês
Pubblicazione: BMJ Publishing Group 2021
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8237187/
https://ncbi.nlm.nih.gov/pubmed/33139350
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/archdischild-2020-319040
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