Oculopharyngeal muscular dystrophy or OPMD is a rare progressive muscle-wasting disease caused by a mutation in a gene called PABPN1 (poly(A)-binding protein nuclear 1), for which there is currently no effective drug therapy. The disease is characterized by swallowing difficulties (dysphagia), limb weakness, and eyelid drooping (ptosis). Dysphagia worsens over time and can lead to chronic choking, regurgitation, aspiration pneumonia, and in severe cases, death.
The company we are profiling today is Australia-based Benitec Biopharma Inc. (BNTC), which is developing a treatment for OPMD.
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