Survey For Young People with Neuromuscular Conditions About Fatigue

03 December 2021 / Posted in: Information

A trainee clinical psychologist at the University of Bath is running this survey about fatigue for young people in the UK (aged 10-24 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.

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Scholar Rock Announces Design of Phase 3 Clinical Trial Evaluating Apitegromab

02 December 2021 / Posted in: Treatments & Research

Scholar Rock have released a community update announcing the design of SAPPHIRE - a clinical trial to further evaluate the safety and efficacy of apitegromab in those who are non-ambulatory and who have SMA Type 2 or Type 3.

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Study with Caregivers of People with SMA

23 November 2021 / Posted in: Information

Evolve is running this study, recruiting caregivers of people with Spinal Muscular Atrophy (SMA) Types 1, 2 or 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.

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NICE, NHS and Roche Agree a 3-year Managed Access Agreement for Risdiplam for SMA Types 1, 2 or 3

19 November 2021 / Posted in: Treatments & Research

We’re absolutely delighted that this third treatment for SMA will now be possible in England (via an MAA), and that the hard work and advocacy for access by SMA UK, MDUK, TreatSMA, all the SMA Community and clinicians has paid off.

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MANATEE Trial Study Centres in the UK Announced

15 November 2021 / Posted in: Treatments & Research

This new global Phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth, in combination with risdiplam in SMA, is due to start in February 2022. UK Centres taking part are Birmingham, GOSH and Oxford.

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Research Project: Participation of Children in the Decisions about their Health

15 November 2021 / Posted in: Information

Radoš Keravica is conducting this PhD research project at the Centre of Disability Studies, University of Leeds, on disabled children’s participation in healthcare decision-making. All participants will receive a £20 shopping voucher as a thank you for their time.

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Zolgensma - 6 Months On

12 November 2021 / Posted in: Treatments & Research

In this webinar, recorded recently, Imelda Hughes (Paediatric Neurology Consultant), Gary McCullagh (Paediatric Neurology Consultant) and Fiona Marley (NHS England) talk to Liz Ryburn, SMA UK’s Support Team Manager, about Zolgensma treatment and what's been happening in the past six months since treatment began in England.

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UK Rare Diseases Framework Questionnaire

09 November 2021 / Posted in: Information

The aim of this questionnaire from the Department of Health & Social Care is to gather feedback from the community on the draft actions that have been developed for inclusion in England’s 2022 action plan for the UK Rare Diseases Framework. Closes 5pm on 26th November.

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We're Hiring! Support Services Lead Job Role

02 November 2021 / Posted in: Information

We’re looking for a Support Services Lead! The successful candidate for this exciting role will lead the delivery of our Support Service workstreams that assist families & adults affected by SMA, and the professionals who support them. Applications close 30th November.

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What’s Happening About Newborn Screening for SMA in the UK?

28 October 2021 / Posted in: Information, Treatments & Research, Support

The short answer is there is a lot of ‘behind the scenes’ activity and that SMA UK is very much part of this. You can expect to hear more in the very near future. In the meantime, we've put together this brief summary which updates you on the activity so far.

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