Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome | Zendy

Guilherme Baldo | Zendy; Fabiano de Oliveira Poswar | Zendy; Andressa Federhen | Zendy; Camila Matzenbacher Bittar | Zendy; Rejane Gus | Zendy; Fernanda Bender | Zendy; Roberto Giugliani | Zendy

Open Access

Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome

Author(s) -

Guilherme Baldo,

Fabiano de Oliveira Poswar,

Andressa Federhen,

Camila Matzenbacher Bittar,

Rejane Gus,

Fernanda Bender,

Roberto Giugliani

Publication year - 2015

Publication title -

journal of inborn errors of metabolism and screening

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 0.125

H-Index - 5

ISSN - 2326-4594

DOI - 10.1177/2326409814567741

Subject(s) - enzyme replacement therapy , mucopolysaccharidosis type ii , keratan sulfate , medicine , hunter syndrome , mucopolysaccharidosis , glycosaminoglycan , placebo , chondroitin sulfate , pathology , disease , anatomy , alternative medicine

Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is a lysosomal storage disorder caused by a deficient N-acetylgalactosamine-6-sulfate sulfatase activity, leading to cellular storage of undegraded keratan sulfate. Recently enzyme replacement therapy (ERT) was approved for MPS IVA, but some of ERT effects are still unknown. In the present study, we aimed to evaluate the efficacy of elosulfase alfa upon glycosaminoglycan (GAG) storage in peripheral blood white blood cells of patients with MPS IVA treated for 6 months, comparing samples from patients who received weekly infusions of enzyme (ERT-W) versus infusions every other week (ERT-EOW) versus placebo. A significant reduction in GAG storage was observed in both ERT-treated groups, with weekly ERT showing slightly better performance than ERT-EOW

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