Glucosylsphingosine (Lyso-Gb

Gaucher disease biomarkers chitotriosidase glucosylsphingosine long-term treatment lyso-Gb1

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
29 Nov 2022
Historique:
received: 04 11 2022
revised: 22 11 2022
accepted: 23 11 2022
entrez: 11 12 2022
pubmed: 12 12 2022
medline: 15 12 2022
Statut: epublish

Résumé

Historically, disease burden and treatment responses in patients with Gaucher disease (GD) was assessed by monitoring clinical data, laboratory, imaging, chitotriosidase (CHITO), and other biomarkers; however, these biomarkers lack specificity and CHITO is uninformative in patients heterozygous or homozygous for the

Identifiants

pubmed: 36499264
pii: ijms232314938
doi: 10.3390/ijms232314938
pmc: PMC9736277
pii:
doi:

Substances chimiques

sphingosyl beta-glucoside 52050-17-6
Psychosine 2238-90-6
Biomarkers 0

Types de publication

Case Reports

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Takeda
ID : IIR-USA 001544

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Auteurs

Matthew M Gayed (MM)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Seung-Hye Jung (SH)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Erin Huggins (E)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Eleanor Rodriguez-Rassi (E)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Stephanie DeArmey (S)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Priya Sunil Kishnani (PS)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Ashlee R Stiles (AR)

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Biochemical Genetics Laboratory, Duke University Health System, Durham, NC 27713, USA.

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Classifications MeSH