On our 8th Day of Christmas, we hear all about Mila, who is the daughter of one of our Trustees.
Mila was born a healthy and beautiful baby. We were thrilled as we had experienced several miscarriages while trying for our second child. I always wanted to have two daughters, Gil didn’t really mind so I felt really grateful and complete. Mila’s arrival was a blessing.
Ionis Pharmaceuticals announced it has initiated a pivotal clinical study of ION373 in patients with Alexander disease, a rare and generally fatal neurological disorder.
Ionis today has announced that they have commenced a pivotal clinical study of ION373 – an investigational antisense medicine designed to reduce the level of GFAP – in patients with Alexander disease.
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.