Sanfilippo Syndrome (MPS)
Sanfilippo Syndrome, or mucopolysaccharidosis III (MPS-III), is a rare, inherited metabolism disorder in which the body cannot properly break down long chains of sugar molecules called glycosaminoglycans leading to progressive degeneration of the central nervous system. Sanfilippo belongs to a group of diseases called mucopolysaccharidoses (MPS). At present there is no cure for Sanfilippo.
Note: As science advances, we continue to experience a fast-evolving landscape of potential new therapies. The families portrayed here offer perspective on coping with rare and/or life-limiting conditions. They also each represent a particular moment in time.
If you are considering a clinical trial for your child, please also see the Courageous Parents Network Guided Pathway on Clinical Trials.