Rationale and Design for a Phase 1 Study of
ManNAc
glomerular hyposialylation
lectin histochemistry
podocytopathy
sialic acid
Journal
Kidney international reports
ISSN: 2468-0249
Titre abrégé: Kidney Int Rep
Pays: United States
ID NLM: 101684752
Informations de publication
Date de publication:
Oct 2019
Oct 2019
Historique:
received:
15
04
2019
revised:
12
06
2019
accepted:
17
06
2019
entrez:
9
11
2019
pubmed:
9
11
2019
medline:
9
11
2019
Statut:
epublish
Résumé
Sialic acids are important contributors to the polyanionic component of the glomerular filtration barrier, which regulates permeability selectivity. Pathologic glomerular hyposialylation, associated with podocyte effacement, has been implicated in human and mouse glomerulopathies. Oral treatment with Here we explore the sialylation status of kidney biopsies obtained from 27 subjects with various glomerular diseases using lectin histochemistry. We identified severe glomerular hyposialylation in 26% of the biopsies. These preliminary findings suggest that this condition may occur relatively frequently and may be a novel target for therapy. We describe the background, rationale, and design of a phase 1 study to test safety, tolerability, and pharmacokinetics of ManNAc in subjects with primary podocyte diseases. We recently demonstrated that ManNAc was safe and well tolerated in a first-in-human phase 1 study in subjects with UDP-
Identifiants
pubmed: 31701055
doi: 10.1016/j.ekir.2019.06.012
pii: S2468-0249(19)31395-6
pmc: PMC6829193
doi:
Types de publication
Journal Article
Langues
eng
Pagination
1454-1462Références
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