Search results for "Morquio syndrome"

Sub-analysis of long-term elosulfase alfa treatment outcomes in adults with Morquio syndrome type A

2017

Burden of disease suffered by patients with Morquio syndrome type A: results from a patient-reported outcomes survey

2014

Burden of disease suffered by caregivers of patients with Morquio syndrome type A: results from a self-reported outcomes survey

2014

Cumulative incidence rates of the mucopolysaccharidoses in Germany

2005

In order to estimate the cumulative incidence rates of the mucopolysaccharidoses (MPS) in Germany, a retrospective epidemiological survey covering the period between 1980 and 1995 was implemented. Multiple ascertainment sources were used to identify affected patients. A prevalence of approximately 0.69 cases per 100,000 births was obtained for MPS I (Hurler phenotype). Within the study period, 4 patients with Hurler/Scheie phenotype and 7 cases with Scheie disease were detected. The cumulative incidence for MPS II (Hunter syndrome) was estimated as 0.64 cases per 100,000 births (1.3 cases per 100,000 male live births); that for MPS III (Sanfilippo syndrome types A, B and C) as 1.57 cases in…

A multi-national, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of BMN 110 treatment for mucopolysaccharidos…

2013

The heart and cardiovascular system in patients with Morquio syndrome type A

2014