Sanfilippo Syndrome (MPS)
Sanfilippo Syndrome, or mucopolysaccharidosis III (MPS-III), is a rare, inherited metabolic disorder belonging to a group of diseases called mucopolysaccharidoses (MPS). There are 4 subtypes of Sanfilippo Syndrome. With Sanfilippo, the body cannot properly break down long chains of sugar molecules called glycosaminoglycans. It is a progressive disorder that primarily affects the brain and central nervous system. There is currently no cure or treatment for Sanfilippo Syndrome. There are several ongoing trials.
Note: As science advances, we continue to experience a fast-evolving landscape of potential new therapies. The families portrayed here offer a 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.