Are you sitting comfortably…
Before finding this product, the only seating my daughter had was a big clumpy tumble form chair and her Wizzybug.
Novartis announces FDA filing acceptance and Priority Review of AVXS-101
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
Another letter to speed up the process
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 receives orphan designation in the EU
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.
Delays in access to treatment: The SMA Community sends letters to key stakeholders
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.
NICE shares an update on Spinraza appraisal
Today we received an update from the NICE on the Spinraza appraisal.
Biogen statement re. EAP in England, Wales and Northern Ireland
In response to our request, Biogen has provided the following update for the SMA Community on the Expanded Access Programme to nusinersen:
SMA to become a terminal condition again in England and Wales
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.