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.
Julian Sturdy MP asks the Health Secretary about Spinraza
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.
Final appraisal meeting for Spinraza (nusinersen)
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.
TreatSMA Families make final plea inside and outside NICE
Vigil held outside NICE whilst others inside make last desperate fight for treatment.