Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline

After Jenny Pearson’s daughter Elise first had a seizure at 16 months old, an MRI came back clean—her brain “looked beautiful,” Pearson recalled to Fierce. But a follow-up scan a year later, just days before Thanksgiving, showed her frontal lobe was now awash in white splotches. A genetic test the week before Christmas gave a troubling diagnosis: Alexander disease.