– Evrysdi is the first and only at-home administered treatment for patients living with SMA – Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date PTC Therapeutics, Inc. announced on May 31st 2022 that the US Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to…
Category: SMA
New Three-Year Data for Genentech’s Evrysdi (risdiplam)
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…
SUNFISH Pivotal Results for Risdiplam (RG7916) Demonstrate Medically Meaningful Benefit in Broadest Age Group of Patients Studied with Type 2 and 3 Spinal Muscular Atrophy
Industry news from PTC therapeutics
Invitation to submit Spinraza® (Nusinersen) to NICE for single technology appraisal
News from Biogen regarding Spinraza® for Spinal Muscular Atrophy
