MPS III Sanifilippo 

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.

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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 

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.

 

We are the only registered charity providing professional support to individuals and families affected by MPS, Fabry or a related disease in the UK.

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MPS Society

MPS House, Repton Place

White Lion Road, Amersham

Buckinghamshire, HP7 9LP

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Registered Charity No.1143472 & SCO41012. Registered Company No. 7726882.

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© 2019 Society for Mucopolysaccharide Diseases