Jonahs Just Begun – Foundation to Cure Sanfilippo, Inc.

The Jonahs Just Begun – Foundation to Cure Sanfilippo, Inc. is a voluntary, non-profit organization whose mission is to educate the public and health care professionals about Sanfilippo syndrome (mucopolysaccharidoses type III or MPS type III), raise funds for research that will hasten the diagnosis of children with Sanfilippo syndrome, recruit patients to participate in their natural history study, and raise funds for treatment research. MPS disorders are a group of rare genetic disorders caused by a deficiency of one of the lysosomal enzymes. Sanfilippo syndrome is one of seven MPS disorders and has been subdivided into four subgroups: MPS-III types A, B, C and D. Disease severity and age of onset vary by type, but all are associated with a progressive loss of speech, motor and cognitive function.