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Jazz Pharmaceuticals presents new findings from caregiver survey on outcomes of Epidiolex® (cannabidiol) for adult and pediatric patients with severe, childhood-onset epilepsies

Jazz Pharmaceuticals plc announced new real-world data from the BECOME (BEhavior, COgnition, and More with Epidiolex) Caregiver Survey that showed a substantial proportion of caregivers of adult and pediatric patients with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS) prescribed Epidiolex (cannabidiol) oral solution reported improvements in patients’ seizure and non-seizure outcomes across all age groups.


Ionis announces European Medicines Agency accepts Marketing Authorization Application of tofersen to treat rare, genetic form of ALS

Ionis Pharmaceuticals Inc. announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for review of tofersen, an investigational medicine for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). SOD1-ALS is a progressive and uniformly fatal disease that affects fewer than 1,000 people across Europe.