Category Archives: News
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.
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, …
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 …
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 …
Shire announces 2013 BRAVE Awards recognizing caregivers – Nominations accepted through June 30, 2013
The Shire BRAVE Awards program is an initiative of Shire plc, a global specialty biopharmaceutical company, to acknowledge ordinary people who give of themselves by caring for others in a …
Hunter In Focus Exhibit
Hunter in Focus, a Positive Exposure™ Project, is the first global photography exhibit to raise awareness for Hunter syndrome, a rare and progressive genetic disorder. This exhibit shares the stories …
Give Your Child the Gift of Summer Camp!
Summer is not far away and camp can be the highlight of a child’s summer experience. Camping creates a connection to people and experiences that are not a part of …
BioMarin Submits Vimizim BLA to the U.S. FDA for the Treatment of MPS IVA
BioMarin Pharmaceutical Inc. announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under …
Genzyme Scholarship Program – Apply by April 30
What Is Chart Your Own Course? Genzyme has introduced Chart Your Own Course, a new program established to help students in the lysosomal storage disorder (LSD) community pursue their educational …
Shire MPS II Neurodevelopmental Observational Study
Shire Human Genetic Therapies, Inc. is sponsoring an observational clinical trial to evaluate the neurodevelopmental status of pediatric patients with MPS II who may show signs of central nervous system …
