Treatment Efficiency in Gaucher Patients Can Reliably Be Monitored by Quantification of Lyso-Gb1 Concentrations in Dried Blood Spots

Treatment Efficiency in Gaucher Patients Can Reliably Be Monitored by Quantification of Lyso-Gb1 Concentrations in Dried Blood Spots

Gaucher disease (GD) is a lysosomal storage disorder that responds well to enzyme replacement therapy (ERT). Certain laboratory parameters, including blood concentration of glucosylsphingosine (Lyso-Gb1), the lyso-derivate of the common glycolipid glucocerebroside, correlate with clinical improvemen...

Full description

Bibliographic Details
Main Authors: Claudia Cozma, Paskal Cullufi, Guido Kramp, Marina Hovakimyan, Virtut Velmishi, Agim Gjikopulli, Sonila Tomori, Steffen Fischer, Sebastian Oppermann, Ulrike Grittner, Peter Bauer, Christian Beetz, Arndt Rolfs
Format: Article
Language:English
Published: MDPI AG 2020-06-01
Series:International Journal of Molecular Sciences
Subjects:
biomarker
enzyme replacement therapy
Gaucher disease
Lyso-Gb1
monitoring
Online Access:https://www.mdpi.com/1422-0067/21/13/4577

Internet

https://www.mdpi.com/1422-0067/21/13/4577

Similar Items