| Recruiting | 1 | 10 | US | Aldurazyme (laronidase), Elaprase (idursulfase), Vimizim (elosulfase alfa), Naglazyme (galsulfase), Mepsevii (vestronidase alfa-vjbk), Lumizyme (alglucosidase alfa), Kanuma (sebelipase alfa) | University of California, San Francisco, Duke University | MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease | 07/31 | 07/32 | | |
NCT02097251: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient |
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| No Longer Available | N/A | | US | UX003, recombinant human beta glucuronidase, rhGUS | Joyce Fox, Ultragenyx Pharmaceutical Inc | Mucopolysaccharidosis Type 7 | | | | |
| Available | N/A | | NA | Mepsevii, UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa | Ultragenyx Pharmaceutical Inc | MPS VII, Mucopolysaccharidosis VII, Sly Syndrome | | | | |