A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children
A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia and premature death.
We are using a combination of gene therapy, stem cells and small molecules to restore metabolic defects in the patient's brain cells. First results in the mouse models of the disease are very encouraging."
Dr. Alexey Pshezhetsky, Professor at CHU Ste-Justine and Lead GlycoNet Investigator
Sanfilippo syndrome belongs to a group of rare diseases known as lysosomal storage disorders.

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