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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...
Autores principales: | Giugliani, Roberto, Muschol, Nicole, Keenan, Hillary A., Dant, Mark, Muenzer, Joseph |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BMJ Publishing Group
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8237187/ https://www.ncbi.nlm.nih.gov/pubmed/33139350 http://dx.doi.org/10.1136/archdischild-2020-319040 |
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