The European Medicines Agency has given orphan drug designation to YH35995, Yuhan Corporation‘s investigational oral therapy for Gaucher disease, following a similar decision from the U.S. Food and Drug Administration in April. Orphan drug designation is granted to medicines for rare diseases, defined in the European Union as…
Levels of the Gaucher disease biomarker glucosylsphingosine (lyso-Gb1) can have different trajectories in people who aren’t actively receiving treatment, a study found. While most untreated patients showed relatively little change in lyso-Gb1 over time, more than one-third experienced increases in the biomarker, which weren’t always associated with immediate disease…
Adults with Gaucher disease type 1 may develop subtle signs of heart dysfunction long before they experience symptoms or show abnormalities on standard heart tests, according to a small new study from Turkey. The researchers found that an advanced heart imaging scan — an ultrasound technique called speckle-tracking echocardiography,…
