Today the entire SMA community is awaiting the outcome of the Committee meeting which is taking place at the National Institute of Health and Care Excellence in Manchester. The Committee is to decide whether spinal muscular atrophy will be treated on the NHS.
Avexis has informed today that the company has submitted applications for regulatory approval of AVXS-101, the groundbreaking gene therapy product developed to treat spinal muscular atrophy.
Submissions have been filed with the European Medicines Agency, the US Food and Drug Administration, and the Japanese regulator.
The National Institute of Health and Care Excellence has begun procedures leading to appraisal of AVXS-101, the first gene therapy to treat spinal muscular atrophy.
Nurture Study reveals new and positive data showing benefits of treating pre-symptomatic infants with Spinraza at the annual congress of the World Muscle Society.
Roche today announced interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam as a treatment for spinal muscular atrophy. The data were presented yesterday at the 23rd International Annual Congress of the World Muscle Society in Mendoza, Argentina.
At the request from TreatSMA, on Monday 24 September five members of TreatSMA held a meeting with Roche in London. We discussed our advocacy work and general wider issues facing the SMA Community.
Today is SMA Awareness Day.
We are excited to share the news that a clinical trial of AVXS-101, the first experimental genetic treatment for spinal muscular atrophy, commenced in London this week.