Developing Resources About Starting Nursery / School – Can You Help?

13 August 2021 / Posted in: Information, Support

We’ve been talking with a clinical nurse specialist who supports many families, about working together with parents / carers to create some information resources for parents and schools. We want to hear from anyone whose child is already at nursery / school, or who is preparing to go. Please register your interest by end of September.

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Children's Heart & Lung Services: Seeking Your Views

13 August 2021 / Posted in: Information

Patients with a heart or lung (or breathing) condition, their carers and families, are being invited to help explore ways that might improve and provide heart and lung care in the future.

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Open Inclusion: New Survey

09 August 2021 / Posted in: Information, Support

Open Inclusion provide services in market research, user insight, innovation and service design. They've recently launched a survey, which closes 20th August, from a global technology company wishing to learn about disabled viewers experiences of online video and television / streaming platforms.

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Updates on Covid vaccinations for children aged over 12 years

04 August 2021 / Posted in: Information, Support

The government has updated its Green Book guidance with more detailed information about which children aged 12-15 years are eligible for a Covid vaccine. The Scottish government has also given more detail about its vaccination programme for those aged 12 - 17 years.

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Appraisal of risdiplam in Scotland delayed further

03 August 2021 / Posted in: Treatments & Research

SMA UK, MDUK and TreatSMA have today written to the Scottish Medicines Consortium (SMC) to raise their concerns about the further delays with processing Roche’s submission for risdiplam.

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Data for Evrysdi Published in NEJM shows Significant Improvement in Survival and Motor Milestones in Babies with SMA Type 1

30 July 2021 / Posted in: Treatments & Research

The study, evaluating the efficacy and safety of Evrysdi™ (risdiplam) in babies aged 1-7 months old with symptomatic SMA Type 1, met its primary endpoint with 29% of infants sitting without support for at least five seconds by month 12 - a milestone not seen in the natural course of the condition.

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Respond to the New National Disability Strategy Proposal

28 July 2021 / Posted in: Information, Support

Today, the Government has launched a new National Disability Strategy. Whilst we welcome this response, we share the concerns being expressed by the Disability Community Alliances and groups. We will be carefully reviewing what is recommended and will work again with our Young Adults' Network and the alliances and groups we belong to, to respond to the Government’s Consultation Survey. You may also want to share your own views - the survey is open until 11th October.

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Survey: Measuring the Family Impact of a Person's Health Condition

22 July 2021 / Posted in: Information, Support

SMA can affect the lives of partners and family members, but this important impact is often ignored. Researchers at Cardiff University have created a simple questionnaire to measure this impact. They need your help to test this questionnaire and are inviting people with SMA and their partners or other family members (aged at least 18 years) to complete this 5-minute survey.

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Community Update: Novartis Discontinues Development of Branaplam

21 July 2021 / Posted in: Treatments & Research

Novartis have confirmed that they've made the difficult decision to discontinue the development of branaplam (also known as LMI070) for the treatment of SMA. They explain that their decision was "made as the result of rapid advancements in the SMA treatment landscape in recent years."

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JCVI Issues Advice on COVID-19 Vaccination of Children and Young People

20 July 2021 / Posted in: Information, Treatments & Research, Support

The JCVI has looked at the available evidence around vaccinating children and young people under the age of 18. They have now advised that children at increased risk of serious COVID-19 disease are offered the Pfizer-BioNTech vaccine. That includes children aged 12 to 15 with severe neurodisabilities, Down’s syndrome, immunosuppression and multiple or severe learning disabilities.

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