Author: Andi Thornton
Nusinersen patient Q&As
Patient organisations have asked for NICE and NHS to answer a few very important questions with regards to MAA and access to Nusinersen. We are now able to share their answers in the attached documents. We will continue to push for expanded access for this treatment to make sure that everybody gets a chance.
AveXis gets EC approval for Zolgensma
AveXis, a Novartis company, announced today that the European Commission (EC) have granted conditional approval for Zolgensma for the treatment of patients with 5q SMA, activating a ‘Day One’ access programme.
Tom Herbert from C4’s hit series ‘Fabulous Baker Brothers’ to judge TreatSMA bake off
For the week commencing 18th May 2020 TreatSMA want to see your creative baking skills. Entries will be judged on decoration and the look of the cake when cut. There is also a 3rd category for creativity if you are unable to get traditional ingredients.
Tom Herbert from C4’s hit series ‘Fabulous Baker Brothers’ to judge TreatSMA bake off
For the week commencing 18th May 2020 TreatSMA want to see your creative baking skills. Entries will be judged on decoration and the look of the cake when cut. There is also a 3rd category for creativity if you are unable to get traditional ingredients.
Roche submit EAMS paperwork for early access program
We have pleasure in announcing the exciting news that Roche today submitted their application for an Early Access to Medicines Scheme (EAMS), a UK specific early access program which is approved by the MHRA.
Roche submit EAMS paperwork for early access program
We have pleasure in announcing the exciting news that Roche today submitted their application for an Early Access to Medicines Scheme (EAMS), a UK specific early access program which is approved by the MHRA.
Roche posts Professor Servais update on FIREFISH in infants with SMA
Key findings: Significant finding that infants were sitting greater than 5 seconds after a year on treatment. A significant improvement in motor function children achieved unexpected milestones of sitting or even standing majority of children maintained ability to swallow
Roche posts Professor Servais update on FIREFISH in infants with SMA
Key findings: Significant finding that infants were sitting greater than 5 seconds after a year on treatment. A significant improvement in motor function children achieved unexpected milestones of sitting or even standing majority of children maintained ability to swallow