MPS III Sanfilippo 

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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 

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MPSIIIA trial

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.