Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-a-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIB

Authors :: Whitley CB
Vijay S
Yao B
Pineda M
Parker GJM
Rojas-Caro S
Zhang X
Dai Y
Cinar A
Bubb G
Patki KC
Escolar ML
Source :: MOLECULAR GENETICS AND METABOLISM, r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu, instname, r-FSJD: Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Publication Year :: 2019
Publisher :: ACADEMIC PRESS INC ELSEVIER SCIENCE, 2019.
Abstract: Mucopolysaccharidosis IIIB is caused by a marked decrease in N-acetyl-a-d-glucosaminidase (NAGLU) enzyme activity, which leads to the accumulation of heparan sulfate in key organs, progressive brain atrophy, and neurocognitive decline. In this open-label study, 11 eligible patients aged 2 to

Subjects :: SBC-103
Enzyme replacement therapy
Mucopolysaccharidosis
Lysosomal storage disease
Sanfilippo syndrome
Heparan sulfate

ISSN :: 10967192
Database :: OpenAIRE
Journal :: MOLECULAR GENETICS AND METABOLISM, r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu, instname, r-FSJD: Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
Accession number :: edsair.dedup.wf.001..7bcf7cf116353a82256c4556134b914c

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