Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome
J. inborn errors metab. screen
; 3: e140010, 2015. graf
Article
in English
|
LILACS-Express
| LILACS
| ID: biblio-1090871
Responsible library:
BR1.1
ABSTRACT
Abstract 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.
Full text:
Available
Collection:
International databases
Database:
LILACS
Type of study:
Controlled clinical trial
Language:
English
Journal:
J. inborn errors metab. screen
Journal subject:
Medicina Cl¡nica
/
Patologia
Year:
2015
Document type:
Article
Affiliation country:
Brazil
Institution/Affiliation country:
Hospital de Clínicas de Porto Alegre/BR
/
Hospital de Clínicas de Porto alegre/BR