Monthly Archives: May 2013

Ultragenyx today announced a Phase 1/2 study of UX003 for MPS VII

Ultragenyx announced a Phase 1/2 study of UX003 for MPS VII. Read the full press release. Read the Q&A with Dr. Emil Kakkis.

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May 15 is International MPS Awareness Day!

On MPS Awareness Day we celebrate our MPS family and spread awareness of these diseases. We also honor Dr. Emil Kakkis, who developed Aldurazyme®, the enzyme replacement therapy for MPS I, and whose company, Ultragenyx, announced on MPS Awareness Day that a Phase 1/2 study of UX003 for MPS VII.

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Synageva BioPharma joins the International MPS Network in support of MPS Awareness Day 2013

Synageva, a biopharmaceutical company developing therapeutic products for rare diseases, joins the International MPS Network and others around the world in observing MPS Awareness Day 2013. During MPS Awareness day, …

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Icahn School of Medicine at Mount Sinai collaboration with bene pharmaChem

Icahn School of Medicine at Mount Sinai announced an agreement with bene pharmaChem GmbH to collaborate on clinical studies for MPS. Research grants to Dr. Calogera Simonaro from the National MPS Society funded her early research with pentosan polysulfate.

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Icahn School of Medicine at Mount Sinai and Bene Pharmachem Gmbh To Collaborate On Clinical Studies for Mucopolysaccharidoses

The Icahn School of Medicine at Mount Sinai has signed an agreement with bene pharmaChem GmbH to collaborate to conduct clinical studies of pentosan polysulfate (PPS) in patients with mucopolysaccharidoses …

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LYSOGENE received US orphan drug designation for SAF-301 for treatment of MPS III A

LYSOGENE announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead gene therapy product SAF-301 for the treatment of MPS III A. SAF-301 …

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