Lyso-Gb1 Biomarker May Be Useful for Monitoring Disease Activity in Children, Study Suggests

Glucosylsphingosine (lyso-Gb1) seems to be a useful biomarker for monitoring disease activity in children with Gaucher disease, helping physicians determine when enzyme replacement therapy should be initiated, a study suggests. The study, “Glucosylsphingosine (lyso-Gb1) as a Biomarker for Monitoring Treated and Untreated Children with Gaucher Disease,” was…

Cerdelga Continuously Provides Long-term Benefits for Gaucher Patients, Study Shows

Long-term treatment with Cerdelga (eliglustat), a substrate reduction therapy for Gaucher disease type 1, is safe and continuously improves blood parameters, decreases spleen and liver volumes, and reduces markers of disease activity, results from an eight-year study show. The study, “Outcomes after 8 Years…