Positive News for GOSH children with SMA Type 1
01 June 2017
Families with eligible children with SMA Type 1 currently under Great Ormond St Hospital’s care, who wish to access the nusinersen EAP, will be able to do so over the next 3 months. This includes children under GOSH’s care who currently access treatment in Europe.
In answer to our query on 2nd June, GOSH further clarified:
“GOSH can only provide the nusinersen EAP to children who are under GOSH clinical care currently. I regret that we cannot take referrals from out of the GOSH UK catchment area and other countries, as we have not been provided with any extra resources. The health authorities in those regions and countries are responsible for provision of the nusinersen EAP treatment to children resident in the areas.”
“Also, with yesterday's announcement of European Commission (EC) granting a marketing authorization for SPINRAZA® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), there will now be an onus on the individual European countries to provide this treatment for the SMA children.”
Our campaign with MDUK, the SMA Trust and TreatSMA for access to the nusinersen SMA Type 1 EAP for all UK children wherever they live, continues.