NOVATO, Calif., Sept. 26, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that three abstracts related to UX001 Sialic Acid for Hereditary Inclusion Body Myopathy, also known as GNE Myopathy, were accepted for poster presentation at the 18th Annual World Muscle Society Congress, October 1-5, 2013 in Asilomar, California.
The following abstracts will be presented as posters on October 2nd from 2:30 pm to 4:00 pm, in the Distal Myopathies Session:
- GNE Myopathy Functional Activity Scale (GNEM-FAS): Development of a Disease-Specific Instrument for Measuring Function and Independence;
- Characterization of Strength and Function in Adults with Inclusion Body Myopathy (HIBM)/GNE Myopathy;
- A Prodrug Strategy to Increase Oral Absorption and Bioavailability of Sialic Acid in an HIBM Mouse Model
These abstracts are available on the Ultragenyx website, www.ultragenyx.com.
About HIBM
HIBM has recently been renamed GNE myopathy, but historically has also been called Quadriceps Sparing Myopathy (QSM), Inclusion Body Myopathy type 2, Distal Myopathy with Rimmed Vacuoles (DMRV) and Nonaka myopathy. HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a defect in the biosynthetic pathway for sialic acid (SA). Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. The body's failure to produce enough sialic acid causes muscles to slowly waste away and can lead to very severe disability within 10 to 20 years of diagnosis, with patients often ending up wheelchair-bound within that time. There is currently no approved therapy.
About Ultragenyx
Ultragenyx is a privately held, clinical-stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.