2023-06-24 14:00:00
A promising treatment for Gaucher disease
According a recent study published in JAMA Network Openambroxol, a drug so far commonly used for coughs, could be effective in treating Gaucher’s disease, a rare genetic pathology which notably causes enlargement of the spleen and liver.
To achieve these results, the researchers administered ambroxol to 28 patients with Gaucher’s disease for six months. At the end of the study, significant improvements were seen in 26 of the 28 patients. “Patients treated with ambroxol showed improvements in hematology, visceral volumes and plasma biomarkers”, say the scientists.
However, further studies will be needed to determine whether ambroxol works over the long term and to fully assess its effectiveness as a treatment for Gaucher disease.
Understanding Gaucher Disease
Gaucher’s disease is a rare genetic disease characterized by the abnormal accumulation of a substance called glucocerebroside in cells and organs. This buildup causes symptoms such as enlarged liver and spleen, brown pigmentation of the skin, fatigue and, in severe cases, liver failure or kidney failure.
The disease is usually diagnosed in children but can also appear in adults.
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