Vanda pharmaceuticals announces first patient dosed in a trial evaluating vca-894a in charcot-marie-tooth disease type 2s

Washington , june 10, 2025 /prnewswire/ -- vanda pharmaceuticals inc. (vanda) (nasdaq: vnda) today announced the first dose in the first-in-human clinical trial to evaluate the safety and tolerability of vca-894a, an antisense oligonucleotide (aso) therapeutic, for a patient with a rare variant in the ighmbp2 gene causing charcot-marie-tooth disease type 2s (cmt2s). vca-894a is being developed for a patient who was first diagnosed at the age of 5 with a rare subtype of cmt disease known as cmt2s.1 cmt2s is an inherited neuromuscular disorder that progressively leads to muscle weakness and loss of motor function, and has an estimated prevalence of less than 1 in 1,000,000 worldwide.2 the severity and clinical presentations of cmt2s are influenced by the diverse genetic variants associated with cmt disease.
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