Informing therapeutic decisions in a rare disorder
Long term follow-up of 103 untreated adult patients with type 1 Gaucher disease
Therapy for Gaucher disease, a rare metabolic disorder, has been available for almost 30 years, but whether all patients should receive the costly treatment has remained controversial. Upon longitudinal analysis of untreated patients, those with mild disease have now been shown to remain clinically stable over a long time. This important finding, which is also reflected by CENTOGENE’s Gaucher-specific biomarker LysoGb1, was published in Journal of Clinical Medicine.