Tonight at midnight not only will October end, but so will the Expanded Access Programme that has saved the lives of nearly 100 children in the UK.
As the Community representatives were taking part in NICE proceedings in Manchester, in the Commons, a Yorkshire MP has questioned the Health Secretary on Spinraza.
TreatSMA along with other patient advocacy groups, patient experts and clinicians, attended and contributed at the final appraisal meeting for Spinraza (nusinersen) at NICE office on Tuesday 23rd October.
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.