Contact - Back to School Advice (England)
28 August 2020 / Posted in: Information, Support
The charity Contact for Families with Disabled Children, have published some guidance around this to help answer questions and concerns that parents may have ahead of their children going back to school in September.
Survey Exploring Access to the Standards of Care for People who have SMA in the UK
28 August 2020 / Posted in: Information, Support
The John Walton Muscular Dystrophy Research Centre in Newcastle invites one respondent for each child / adult who has SMA to take part in gathering this important information that will help guide future practice.
Grant Support Available to Families on Lower Incomes in England
28 August 2020 / Posted in: Information, Support
National charity Family Fund, received £10m from the Department for Education to provide extra grants. The grants can be used to buy computers and play equipment as well as household items such as cookers and fridges.
NICE Widens Scope of its Appraisal of Zolgensma
28 August 2020 / Posted in: Treatments & Research
This will now be in line with the European marketing authorisation and will now also include those who have 5qSMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene and, if evidence allows, consideration may be given to a sub-group of people who are pre-symptomatic. This widens the possibilities and is what patient groups have been advocating.
Nice Announces Next Stages in Appraisal of Risdiplam / Evrysdi
28 August 2020 / Posted in: Treatments & Research
When we heard in April this year that NICE’s risdiplam appraisal was to be put on hold due to Covid-19, patient groups wrote jointly to NICE urging them to reinstate the process at the earliest possible time due to the great potential this treatment has for the SMA Community. We were pleased to hear this week that the appraisal is again progressing.
Campaign for Step-Free Access at all Train Stations
24 August 2020 / Posted in: Information, Support
The Step-Free Access Campaign, set up by Ian Cook, is about petitioning for step-free access at all railway stations, and on trains, as so many are still inaccessible for disabled people. Add your voice to the campaign.
About the Young Scientists who Make Up the UK SMA Research Conference’s Committee
24 August 2020 / Posted in: Treatments & Research
Six dedicated young PhD students from institutions across the UK are organising an online SMA research conference on 10th & 11th September. Here, you can find out more about them, their work and their interests outside the laboratory!
FAQs About Roche’s Risdiplam Compassionate Use Programme (CUP) for Those who have SMA Type 1 or 2
21 August 2020 / Posted in: Treatments & Research
These cover who may be eligible for access, the application process and how the treatment programme will work. They also cover some other questions that have been asked about what future access routes to risdiplam there may be for those who have Type 3.
Biogen: Community Update on European / Global Access to Nusinersen
20 August 2020 / Posted in: Treatments & Research
Biogen has provided the latest update on access to nusinersen treatment around the world. There are now 29 European countries that have access to nusinersen via regular reimbursement.
Back to School – Webinar & Online Q&A
18 August 2020 / Posted in: Information, Support
Many parents of children with serious health needs have concerns and unanswered questions about their children returning to school in September. WellChild have organised an online Q&A, on 20th August, with a panel of experts from Department of Education, law, health and education to help answer some of these burning questions.
