MPS III Sanfilippo 


What is MPS III

MPS III, known as Sanfilippo disease, is one of the mucopolysaccharide storage diseases. MPS III was first identified by Dr Sanfilippo in 1963 and includes 4 different types A, B, C and D.

Useful links

For advocacy support

For treatment options

For research and clinical trials

Contact us


Our support line is open 9-5pm

Monday-Friday: 0345 389 9901

Out of hours line: open 5pm-10pm Monday-Friday and weekends:

07712 653 258 



Royal Manchester Children’s Hospital (RMCH) has opened a stem cell gene therapy bone marrow transplant for MPSIIIA (Sanfilippo Syndrome). The trial is open to children with a predicted severe illness course (family history and/or genetic diagnosis) and aged under 2 years. We are open to patients from anywhere in the world, and treatment, travel and accommodation costs are met by the trial.