Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I.
Saved in:
| Title: | Limited data to evaluate real-world effectiveness of enzyme replacement therapy for mucopolysaccharidosis type I. |
|---|---|
| Authors: | Kuiper GA; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands., Nijmeijer SCM; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands., Roelofs MJM; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands., van der Lee JH; Amsterdam UMC, University of Amsterdam, Pediatric Clinical Research Office, Meibergdreef 9, Amsterdam, Netherlands., Hollak CEM; Amsterdam UMC, University of Amsterdam, Endocrinology and Metabolism, Amsterdam Lysosome Center 'Sphinx', Meibergdreef 9, Amsterdam, Netherlands., Bosch AM; Amsterdam UMC, University of Amsterdam, Pediatric Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, Netherlands. |
| Source: | Journal of inherited metabolic disease [J Inherit Metab Dis] 2019 Sep; Vol. 42 (5), pp. 762-775. Date of Electronic Publication: 2019 May 16. |
| Publication Type: | Journal Article; Systematic Review |
| Journal Info: | Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
|
Full text is not displayed to guests.
Login for full access.
|
|
| ISSN: | 1573-2665 |
|---|---|
| DOI: | 10.1002/jimd.12103 |
