Enzyme replacement therapy for the mucopolysaccharide storage disorders.

Enzyme replacement therapy for the mucopolysaccharide storage disorders.

Item
http://www.wikidata.org/entity/Q34628680
The cell biology of disease: lysosomal storage disorders: the cellular impact of lysosomal dysfunctionCNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorderEnzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type miceSialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage diseaseAn overview of Korean patients with mucopolysaccharidosis and collaboration through the Asia Pacific MPS Network.Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseasesThe first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product.Current and potential therapeutic strategies for mucopolysaccharidoses.Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase.Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice.Enzyme replacement therapy and extended newborn screening for mucopolysaccharidoses: opinions of treating physicians.
P2860

Enzyme replacement therapy for the mucopolysaccharide storage disorders.

Item
http://www.wikidata.org/entity/Q34628680
description
2002 nî lūn-bûn
@nan
2002 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
2002 թվականի մայիսին հրատարակված գիտական հոդված
@hy
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
name
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@ast
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@en
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@nl
type
label
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@ast
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@en
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@nl
prefLabel
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@ast
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@en
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@nl
P1433
P1476
P2093
P2860
P304
P31
P356
P407
P433
P478
P577
P698
P921
P356
P1433
P1476
Enzyme replacement therapy for the mucopolysaccharide storage disorders.
@en
P2093
P2860
P304
P31
P356
P407
P433
P478
P577
P698
P921