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, 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.
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.
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.
- 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
TreatSMA are extremely happy and proud to announce that Marni Smyth has become the sixth trustee that has joined our team. Marni has been in discussions with TreatSMA for a number of weeks and we take great pleasure in announcing the news.
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions. This week Marion focused on taping.
Last weekend we announced on our website the changes that Roche were making to their compassion access program in light of Covid-19 challenges. While this was a welcome move we also published a statement questioning the decision not to go further with access. Following that statement we held a call with Roche and their UK Managing Director: We wanted to provide you a summary of that conversation.