We at TreatSMA would love to thank everyone for participating in Saturdays LIVE SMA DAY. We had a whole array of amazing speakers , sports professionals, worldwide known doctors, professionals in SMA and not forgetting those from the community itself. We also had a few visits from the legendary Geordie sausage!
TreatSMA were fortunate enough to be invited to the event along with having a stand for people to come and talk to us about our journey fighting for treatments for rare diseases.
TreatSMA are going live all day on Facebook on the 28th September with a huge event jampacked with speakers covering lots of different aspects of SMA, ranging from physiotherapy to science.
In a recent update we mentioned that hospitals were going to be asked to submit expressions of interest to provide Spinraza for adult patients. We can now confirm that that request has gone out and it is now down to hospitals to submit to NHS England.
Recently there has been a drive from various stakeholders involved in how new medicines or medical devices gain access to the UK market to introduce changes that reflects modern needs of the country. This drive for change generated discussions and…
Today we learn that treatment for other rare diseases were approved for cystic fibrosis in Scotland and Batten disease. Thoughts and congratulations go out to everyone affected by or working hard to receive treatment for these diseases from TreatSMA.
Acaster Lloyd Consulting Ltd, an independent research consultancy specialising in patient research, on behalf of a pharmaceutical company, is conducting an online survey for adults and caregivers of children with a medical diagnosis of spinal muscular atrophy (SMA) to understand views about the importance of different aspects of treatment for SMA and need your help.
Now that the MAA has been released attention turns towards monitoring progress with regards to the implementation of treatment for those currently eligible. Eligible patients should have access to treatment within 90 days of the NICE guidance being released subject to any clinical or commissioning issues. This is what we know so far.