Rare Diseases

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Rare Diseases News & Journal Articles

  • Novel First-in-class Agent Shows Promise in Wilson's Disease In patients with Wilson’s disease, once-daily oral treatment with bis-choline tetrathiomolybdate rapidly lowered free copper levels, which correlated with reduced disability, improved neurological status, and stable liver function in an open-label phase 2 study.
  • ANA Inotersen Shows Efficacy in Hereditary ATTR Improvements in neurologic and quality-of-life endpoints were seen in patients with hereditary transthyretin amyloidosis with polyneuropathy by using the investigational drug inotersen; side effects were called 'monitorable.'

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