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Université de Montréal: Major Breakthrough in Sanfilippo Syndrome Treatment Development

Sanfilippo syndrome: Research team resolves structure of crucial enzyme for the first time

Diabète de type 2 : le rôle d'une protéine montré du doigt

Researchers identify a new mechanism underlying atherosclerosis

Researchers identify a new mechanism underlying atherosclerosis Glycomics researchers at the University of Alberta and CHU Sainte-Justine have reported a discovery that could lead to new treatments for cardiovascular disease. The researchers identified a new mechanism responsible for the buildup of plaque on artery walls, a process known as atherosclerosis. This plaque, made up of fats, cholesterol and other substances, can restrict blood flow and is a major factor in cardiovascular disease. We identified a new mechanism underlying atherosclerosis. We also demonstrated that this can be addressed pharmacologically. Using inhibitors developed in our lab, we found that this could be a new strategy for therapeutics in cardiovascular disease.

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.