One of the questions I never expected to face as a parent is how to explain a chronic illness to a toddler. My son is almost 2 years old, which means he doesn’t understand much about Gaucher disease. He doesn’t know what a genetic disorder is, why I need…
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…
