Vanda pharmaceuticals announces the publication of an article titled "potential aso-based personalized treatment for charcot-marie-tooth disease type 2s"

Washington , feb. 24, 2025 /prnewswire/ -- vanda pharmaceuticals inc. (vanda) (nasdaq: vnda) today announced the publication of an article entitled "potential aso-based personalized treatment for charcot-marie-tooth disease type 2s" in molecular therapy nucleic acids1, a cell press partner journal, that describes the development of a novel antisense oligonucleotide (aso) therapeutic, vca-894a, for a patient with a rare variant in the ighmbp2 gene causing charcot-marie-tooth disease type 2s (cmt2s). in 1886, jean-martin charcot, pierre marie, and henry tooth independently described this hereditary motor and sensory neuropathy in their patients, paving the way for charcot-marie-tooth (cmt) disease research.2,3 it was not until 1968 that a distinction was made in cmt disease, creating the classification of cmt1 and cmt2.4 since then, cmt disease (omim: 616155) has been used as a model disease to describe genetic heterogeneity.5 however, the high genetic and allelic heterogeneity of cmt disease poses a challenge for both diagnosis and treatment.
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