Browsing Paediatrics and Child Health by Subject "Activities of daily living (ADL)"

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Health-related quality of life in mucopolysaccharidosis : looking beyond biomedical issues

Hendriksz, Christian J.; Berger, Kenneth I.; Lampe, Christina; Kircher, Susanne G.; Orchard, Paul J.; Southall, Rebecca; Long, Sarah; Sande, Stephen; Gold, Jeffrey I. (BioMed Central, 2016-08-26)

The mucopolysaccharidoses (MPS) comprise a heterogeneous family of rare, genetic lysosomal storage disorders that result in severe morbidity and reduced life expectancy. Emerging treatments for several of these disorders ...
Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio a syndrome in an open-label, multi-center, phase 3 extension study

Hendriksz, Christian J.; Parini, Rossella; AlSayed, Moeenaldeen D.; Raiman, Julian; Giugliani, Roberto; Mitchell, John J.; Burton, Barbara K.; Guelbert, Norberto; Stewart, Fiona J.; Hughes, Derralynn A.; Matousek, Robert; Hawley, Sara M.; Decker, Celeste; Harmatz, Paul R. (Elsevier, 2018-02)

BACKGROUND : Long-term safety and efficacy of elosulfase alfa enzyme replacement therapy (ERT) were assessed in 173 patients with Morquio A syndrome (mucopolysaccharidosis IVA) in a 96-week, open-label, multi-center, phase ...

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