Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that the European Medicines Agency (EMA) has granted orphan drug designation to ION373 for the treatment of people with Alexander disease, a severe, progressive and debilitating condition that can eventually result in death due to loss of control over autonomic functions like breathing. Infants and young children who develop the condition rarely survive beyond their teenage years or young adulthood.
Link to Press Release : https://ir.ionispharma.com/news-releases/news-release-details/ionis-treatment-alexander-disease-granted-orphan-drug-status-ema?_ga=2.130928783.1638857024.1581593556-1055668296.1574331044
Learn more on our website :
Alexander Disease type 1 & Alexander Disease type 2