Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
Today TreatSMA along with SMA Support UK and Muscular Dystrophy UK sent another letter to NICE, in which we asked that nusinersen be placed on the agenda of the nearest Committee C meeting which will take place on 6 February.
SRK-015, an experimental drug for SMA being developed by the US biotechnology company Scholar Rock, has been awarded the Orphan Medicinal Product designation in the European Union.
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.