Ultragenyx Initiates Novel Disease Monitoring Program for Heredi...

First patient enrolled in integrated program designed to improve knowledge of rare disease

NOVATO, CA -April 8, 2013 -Ultragenyx Pharmaceutical Inc. today announced the launch of a unique Disease Monitoring Program (DMP) for Hereditary Inclusion Body Myopathy (HIBM), also known as GNE Myopathy. The goal of the HIBM-DMP is to improve the body of knowledge about this rare disease and its typical course. This novel program is being conducted in partnership with the University of Newcastle's TREATNMD organization, a global neuromuscular physician network in Newcastle, England.The main objectives of the HIBM-DMP are to expand knowledge of the clinical presentation, progression and variation of HIBM patients; identify and qualify biomarkers and other efficacy measures; inform the design and interpretation of clinical studies of investigational products for HIBM and eventually to optimize patient management.

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