Moving closer to what could be a landmark FDA approval, Ionis Pharmaceuticals in Carlsbad and partner Biogen have announced the latest favorable results for nusinersen, their drug for spinal muscular atrophy.
In a first, nusinersen showed signs of effectiveness in 13 infants genetically predisposed to the incurable and often fatal disease, but who had not yet shown symptoms. This was measured by evaluating motor milestone events such as head control, sitting up, standing and walking. None of those treated babies have died.
