Fran Sverdrup, Ph.D., Research Fellow in the Center for World Health and Medicine, received funding from Ultragenyx to develop a treatment for Facioscapulohumeral Muscular Dystrophy (FSHD), which causes muscle degeneration and afflicts more than 15,000 people in the U.S.
Dr. Sverdrup’s daughter was diagnosed with the disease several years ago, sending him on a quest to discover a treatment, as no therapies were available at the time. This lack of treatments led him to discover a class of compounds that could turn off the inappropriately expressed gene in FSHD patients. Recently, SLU and Ultragenyx signed an agreement to turn Dr. Sverdrup’s discovery into a treatment for FSHD.
Ultragenyx specializes in developing novel treatments for rare and ultra-rare diseases that might otherwise not be studied due to the small number of patients afflicted. The company has had success in turning these types of discoveries into viable treatments for these rare diseases.
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