Results From a 12-Month Open-Label Phase 1/2 Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
J. inborn errors metab. screen
;
6: 170020, 2018. tab
Artículo
en Inglés
|
LILACS-Express
| LILACS
| ID: biblio-1090966
ABSTRACT
Abstract Gaucher disease (GD) is an autosomal recessive lipid storage disorder, caused by deficient activity of the lysosomal enzyme b-glucocerebrosidase, resulting in accumulation of glucocerebroside in tissue macrophages. HGT-GCB-068 was an open-label study designed to explore the efficacy and safety of velaglucerase alfa in children and adolescents with type 3 GD, a neuronopathic form of the disease. Six treatment-naive patients received infusions of velaglucerase alfa every other week at 60 U/kg over 12 months. Velaglucerase alfa demonstrated a favorable tolerability profile, and 1 infusion-related reaction (headache) was the only drug-related adverse event reported. Numerical increases from baseline in hematological parameters and decreases in visceral parameters were seen at 12 months. http//ClinicalTrials.gov identifier NCT01685216.
Texto completo:
Disponible
Índice:
LILACS (Américas)
Idioma:
Inglés
Revista:
J. inborn errors metab. screen
Asunto de la revista:
Medicina Cl¡nica
/
Patologia
Año:
2018
Tipo del documento:
Artículo
País de afiliación:
Egipto
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India
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Suiza
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Túnez
/
Estados Unidos
Institución/País de afiliación:
Ain Shams University Hospital/EG
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Alexandria University Hospital/EG
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Cairo University Hospital/EG
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KEM Hospital Research Centre/IN
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La Rabta Hospital/TN
/
Shire/CH
/
Shire/US
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