Fox, JE, Volpe, L, Bullaro, J, Kakkis, ED, Sly, WS First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient. Mol.
[rarediseases.org]
First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient. Mol Genet Metab. 2015 Feb. 114 (2):203-8. [Medline].
[emedicine.medscape.com]
Multiple assays of other specific treatments are being performed in animal models: allogenic bone marrow transplantation, gene therapy and enzyme replacement therapy (with
[orpha.net]