Mucopolysaccharidosis (MPS): treatment
Evidence-based neurology checklist on mucopolysaccharidosis (mps): treatment: 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
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
References
- Wiśniewska K, Wolski J, Gaffke L, Cyske Z, Pierzynowska K, Węgrzyn G. Misdiagnosis in mucopolysaccharidoses. J Appl Genet 2022; 63:475-495.
- 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.
