AN INNOVATIVE TREATMENT FOR GAUCHER’S DISEASE
Gaucher’s disease is a genetic disorder that affects less than 10,000 people worldwide.
It is characterized by deficiency in glucocerebrosidase (or beta-glucosidase), an enzyme that breaks down certain fat molecules. Accordingly, fat-engorged cells (called Gaucher cells) accumulate in different parts of the body, mainly the spleen, liver and bone marrow. This buildup can cause, among other things, an increase in volume of the spleen and liver, anemia, excessive bleeding, bruising and bone diseases. Cerdelga® developed by Sanofi Genzyme, our specialty care business unit encompassing rare diseases, is designed to partially inhibit enzyme glucosylceramide synthase and, thereby, reduce production of glucosylceramide. It is a new first-line oral treatment for certain adults with the disease.
Francisco, who suffers from Gaucher’s disease, Portugal.
Encouraging prospects in atopic dermatitis.
A KEY DISCOVERY FOR PATIENTS
The US health authority, the Food and Drug Administration (FDA), has granted “breakthrough” designation to dupilumab for the treatment of adults with moderate to severe atopic dermatitis. Atopic dermatitis is a chronic inflammatory disease characterized by inflammation of the skin and itching that can affect various parts of the body. Current therapies are often inadequate and can lead to significant side effects. Dupilumab, co-developed with Regeneron, is intended to block the action of two proteins that appear to play a key role in allergic inflammation. This FDA designation reflects the promising results of phase I and II studies, confirmed by the positive results of two clinical phase III trials.