Monthly Archives: December 2014

28th Annual Family Conference – Disney

Thank you to everyone who attended the National MPS Society’s 28th Annual Family Conference December 18-20, 2014 at Disney!

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Ultragenyx Announces Initiation of Phase 3 Study of Recombinant Human Beta-Glucuronidase in MPS VII

Ultragenyx announced the dosing of the first patient in the pivotal Phase 3 study of recombinant human beta-glucuronidase (rhGUS, UX003) for the treatment of MPS VII. Read the full press …

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Synageva BioPharma announces active Investigational New Drug application for SBC-103 for the treatment of MPS IIIB

Synageva BioPharma announces active Investigational New Drug application for SBC-103 for the treatment of MPS IIIB. The company plans to start enrolling patients with MPS IIIB in a Phase 1/2 …

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ArmaGen announces FDA acceptance of IND application for AGT-182 for the treatment of MPS II

ArmaGen announced that the FDA accepted the IND application for AGT-182 for the treatment of MPS II. A Phase 1 clinical trial is expected to begin in the first quarter …

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FDA grants orphan drug designation for BMN 250 for the treatment of MPS IIIB

BioMarin announced that the FDA granted orphan drug designation for BMN 250 for the treatment of MPS IIIB. They expect to initiate clinical studies with BMN 250 in mid-2015. Read …

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