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Browsing Research Articles (Paediatrics and Child Health) by Subject "Elosulfase alfa"

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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 ...
Recommendations for the management of MPS IVA : systematic evidence- and consensus-based guidance

Akyol, Mehmet Umut; Alden, Tord D.; Amartino, Hernan; Ashworth, Jane; Belani, Kumar; Berger, Kenneth I.; Borgo, Andrea; Braunlin, Elizabeth; Eto, Yoshikatsu; Gold, Jeffrey I.; Jester, Andrea; Jones, Simon A.; Karsli, Cengiz; Mackenzie, William; Marinho, Diane Ruschel; McFadyen, Andrew; McGill, Jim; Mitchell, John J.; Muenzer, Joseph; Okuyama, Torayuki; Orchard, Paul J.; Stevens, Bob; Thomas, Sophie; Walker, Robert; Wynn, Robert; Giugliani, Roberto; Harmatz, Paul; Hendriksz, Christian J.; Scarpa, Maurizio (BioMed Central (BMC), 2019-06)

INTRODUCTION: Mucopolysaccharidosis (MPS) IVA or Morquio A syndrome is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the N-acetylgalactosamine-6-sulfatase (GALNS) enzyme, which impairs ...
Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment

Hendriksz, Christian J.; Santra, Saikat; Jones, Simon A.; Geberhiwot, Tarekegn; Jesaitis, Lynne; Long, Brian; Qi, Yulan; Hawley, Sara M.; Decker, Celeste (Elsevier, 2018-04)

Elosulfase alfa is an enzyme replacement therapy for Morquio A syndrome (mucopolysaccharidosis IVA), a multisystemic progressive lysosomal storage disorder. This report includes the primary treatment outcomes and immunogenicity ...

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Browsing Research Articles (Paediatrics and Child Health) by Subject "Elosulfase alfa"

Browsing Research Articles (Paediatrics and Child Health) by Subject "Elosulfase alfa"

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