UltraGenyx Press Release - Ultragenyx Initiates Novel Disease Mon...

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.

The HIBM-DMP is designed to assist the HIBM communities in several ways, including:

- integration of an online registry capturing patient-reported information, a fully monitored physician-driven natural history study, and potentially any post-approval patient follow-up into a single cohesive program.

- knowledge-sharing gained from the program about HIBM with investigators, physicians and patients in a manner compliant with patient data protection regulations.

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