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.
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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.