Long-term improvements in survival and motor milestones in babies with Type 1 Spinal Muscular Atrophy (SMA) – 91% of infants treated with Evrysdi in the FIREFISH study were still alive at three years – – Infants treated with Evrysdi maintained or continued to improve in measures of motor function, including their ability to sit without…
Category: spinal muscular atrophy
Invitation to submit Spinraza® (Nusinersen) to NICE for single technology appraisal
News from Biogen regarding Spinraza® for Spinal Muscular Atrophy