The untimely closure of the Expanded Access Programme of nusinersen and the continuing delays in having this treatment available on the NHS amount to a death sentence for babies diagnosed with the most severe form of SMA. For hundreds of others, they mean an irreversible muscle function loss. We the families at TreatSMA, just like the wider SMA community, are all incredibly frustrated with the apparent lack of progress while watching our near and dear ones waste.
Today we received an update from the NICE on the Spinraza appraisal.
In response to our request, Biogen has provided the following update for the SMA Community on the Expanded Access Programme to nusinersen:
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.