Fred Hutchinson Cancer Research Center and GlaxoSmithKline PLC (GSK) today announced a partnership to develop therapeutics to treat an inherited form of muscular dystrophy.
The goal of the new agreement is to develop a small-molecule-based medicine to potentially reverse facioscapulohumeral muscular dystrophy, or FSHD, by inhibiting the activity of a protein that is incorrectly expressed by the DUX4 gene in people with the disease. The protein activity is what damages muscle cells and leads to progressive muscle weakness and atrophy in FSHD patients.
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