08 October 2018 / Posted in: Treatments & Research
Genetic Alliance UK has launched a campaign to ensure the UK's continued participation in European Reference Networks (ERNs) following our exit from the European Union.
28 September 2018 / Posted in: Information, Support
A new smartphone app, currently being trialled by four train companies, will be rolled out across Britain in Autumn next year to help with passenger assistance, with the aim of making it easier for customers to book help at stations and get a more consistent and reliable service.
27 September 2018 / Posted in: Treatments & Research
This Phase 3 study called STR1VE-EU, will evaluate the safety and efficacy of a one-time intravenous infusion of AVXS-101 in approximately 30 European infants.
27 September 2018 / Posted in: Information
The Lords Economic Affairs Committee has launched its inquiry into the social care system in England and invites written contributions from anyone.
26 September 2018 / Posted in: Information, Support
Taking place between 9th and 15th October, this awareness week is a collaboration between 60+ charities across the UK to raise awareness about the issues affecting those who have experienced pregnancy loss or death of a baby in the UK.
24 September 2018 / Posted in: Treatments & Research
NICE have announced that the public registration facility to observe the next committee meeting about nusinersen on 23rd October will open on 25th September 2018 on their website.
21 September 2018 / Posted in: Information, Support
The charity Contact for Families of Disabled Children say that they have been inundated with calls about issues surrounding school transport. There are a number of support resources available about this on their website, along with an upcoming live Facebook Q&A, and Midlands workshops.
19 September 2018 / Posted in: Help Us
We can’t wait for this year’s Christmas Jumper Day for SMA on Friday 7th December and we hope you can join us to make this one the biggest yet!
19 September 2018 / Posted in: Treatments & Research
Patient groups and clinicians have been invited to take part in this consultation which closes on 17th October. Treatment topics usually reach what is called ‘the scoping stage’ around the time the company plan to apply for a licence for its drug in the UK or Europe. SMA UK will be submitting a response.
11 September 2018 / Posted in: Treatments & Research
Thanks to those who responded to our survey asking for your views on newborn screening. We concluded that a further urgent review of a newborn screening programme for 5q SMA in the UK is imperative.