Sanfilippo Disease: New treatment for rare inherited disease underway

11 agosto 2014

A child — the first — has been recruited into a new study that aims to evaluate the clinical effectiveness of a treatment for Sanfilippo Disease. The disease is a progressive, genetic and life-threatening disease for which there is currently no effective treatment. The syndrome is diagnosed in childhood, with sufferers experiencing deafness, hyperactivity and behavioral problems, progressive developmental delay, and seizures during the later stages of the condition. The condition is usually fatal in late childhood or early adulthood.