Found: 10
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Mortality in patients with Sanfilippo syndrome.
- Published in:
- 2017
- By:
- Publication type:
- journal article
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.
- Published in:
- 2017
- By:
- Publication type:
- journal article
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document.
- Published in:
- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-015-0253-6
- By:
- Publication type:
- Article
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document.
- Published in:
- 2015
- By:
- Publication type:
- journal article
Burden of disease in patients with Morquio A syndrome: results from an international patientreported outcomes survey.
- Published in:
- Orphanet Journal of Rare Diseases, 2014, v. 9, n. 1, p. 1, doi. 10.1186/1750-1172-9-32
- By:
- Publication type:
- Article
Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey.
- Published in:
- 2014
- By:
- Publication type:
- journal article
Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease.
- Published in:
- 2011
- By:
- Publication type:
- journal article
The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK.
- Published in:
- Orphanet Journal of Rare Diseases, 2008, v. 3, p. 1, doi. 10.1186/1750-1172-3-24
- By:
- Publication type:
- Article
The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK.
- Published in:
- 2008
- By:
- Publication type:
- journal article
Supporting the patient and family with mucopolysaccharidosis disorders: the role of patient organizations.
- Published in:
- Acta Paediatrica, 2007, p. 87, doi. 10.1111/j.1651-2227.2007.00217.x
- By:
- Publication type:
- Article