Mucopolysaccharidosis (MPS): treatment

Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .

Mucopolysaccharidosis (MPS): treatment. Rehabilitation, Surgery. Fully referenced neurology checklist.

Treatments

  • Recombinant enzyme replacement therapy: for MPS I, II, IVA, VI and VII
  • Haematopoietic stem cell transplantation (HSCT): for MPS I, II, IV, VI and VII
  • Rehabilitation
  • Surgery

Investigational treatment

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References

  1. Wiśniewska K, Wolski J, Gaffke L, Cyske Z, Pierzynowska K, Węgrzyn G. Misdiagnosis in mucopolysaccharidoses. J Appl Genet 2022; 63:475-495.
  2. Tardieu M, Zérah M, Gougeon ML, et al. Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial. Lancet Neurol 2017; 16:712-720.

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