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.
Useful links
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For research and clinical trials
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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
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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.
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