Breaking Down Barriers - Rare Pride Project
15 September 2021 / Posted in: Information, Support
Breaking Down Barriers is starting the ‘Rare Pride’ project to seek experiences from those who have a rare condition and are part of the LGBTQIA+ community. Join them on Zoom on Tuesday 28th September at 6pm for their first get together and have your voice heard!
New Project: 28 Days of Rare
13 September 2021 / Posted in: Information, Support
The not-for-profit arts organisation Same But Different are running this new project where they'll be creating some visually striking images and sharing individual experiences to highlight rare conditions. They're keen to hear from anyone affected by SMA, in the UK, who would be interested in getting involved.
Study with Caregivers of People with SMA
03 September 2021 / Posted in: Information
Evolve is running this study, recruiting caregivers of people with Spinal Muscular Atrophy (SMA) Type 3, on behalf of Novartis Gene Therapies. The goal is to collect data regarding the use of resources linked to the management of SMA from the perspective of caregivers, as well as time spent caring for patients and any financial impact. Participants will be paid £50 for their time.
Access to Risdiplam in Scotland: Your Views
02 September 2021 / Posted in: Treatments & Research
The SMC has started its review of whether to recommend risdiplam for funding by the NHS in Scotland. SMA UK and MDUK are wanting our joint submission to reflect the views of people in Scotland who are affected by SMA, including those who are bereaved by SMA, so we’re putting out this short survey with a closing date of Thursday 16th September.
What Does The SMA Community Want From SMA UK?
27 August 2021 / Posted in: Information, Support
We know there are a lot of good groups and charities out there. We’re trying to find out where we fit in and how we can be most useful. Let us know your feedback and suggestions by taking our survey. Closing date: Sunday 12th September
Survey For Young People with Neuromuscular Conditions About Fatigue
20 August 2021 / Posted in: Information
A trainee clinical psychologist at the University of Bath is running this survey about fatigue for young people (age 11-18 years) who have neuromuscular conditions. The survey takes approximately 20-30 minutes to complete and there is also an optional prize draw for participants to enter.
MDUK Muscles Matter 2021 SMA (Paediatric Focus) Seminar
19 August 2021 / Posted in: Information, Treatments & Research
Muscular Dystrophy UK are holding this online seminar on Monday 23rd August, 10am-12pm, to keep you up-to-date with the latest research into SMA. You will have the chance to hear from experts about progress into research and treatments, and get the opportunity to ask questions.
NICE methods, Process and Topic Selection for Health Technology Evaluation: Proposals for Change Consultation Now Open
19 August 2021 / Posted in: Information, Treatments & Research
This eight-week consultation on the final stage of NICE’s methods and processes review is now open for any responses by 13th October. We will attend meetings with partners and alliances to review what is being proposed and join with responses.
Phase 3 Trial to Evaluate OAV-101 Intrathecal (IT) in People with SMA
17 August 2021 / Posted in: Treatments & Research
The FDA in the US has removed the partial clinical trial hold initiated in October 2019 and determined that Novartis Gene Therapies’ investigational compound OAV-101 IT clinical trial for people with SMA may proceed. Novartis now plans to evaluate the clinical efficacy, safety and tolerability of OAV-101 IT in treatment naïve people with SMA who are between two and 18 years of age, able to sit, but have never walked.
Scotland Starts to Progress Review of Whether to Recommend Risdiplam for NHS Funding
17 August 2021 / Posted in: Treatments & Research
We are pleased to see that the Scottish Medicines Consortium has now made this announcement. We and the other patient groups will be making submissions in time for the deadline for these of 4th October 2021.
