Contact: Petition for a Carers COVID-19 Payment

03 June 2020 / Posted in: Information, Support

This payment would help with some of the extra costs many are facing due to the pandemic but would importantly also send a message from Government to unpaid carers that they are valued at this difficult time.

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New Survey from Together For Short Lives

02 June 2020 / Posted in: Information, Support

This survey is designed to gather the experiences and views of parents / carers of children (aged 0-25 years) who have a life-limiting condition, in relation to how COVID-19 is affecting them.

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Three Further Centres will be Offering Nusinersen Treatment to Adults

02 June 2020 / Posted in: Treatments & Research

NHS England has advised us that three more NHS Trusts will offer this service: King's College Hospital, North Bristol and Southampton University Hospital. They will join the other eight centres. Read more here about what is happening with adult services.

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New Survey: Parent’s Seeking Help for, and Care of, a Sick or Unwell Child During COVID-19 ‘Stay at Home’ Period

28 May 2020 / Posted in: Information, Support

The University of Plymouth has launched this survey as it's hoped the results will advance understanding of how these extraordinary times are affecting parents' thinking about, and perception of, access to services for children when they are sick or unwell.

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AveXis UK Updates the Community on Progress Towards Access to Zolgensma

28 May 2020 / Posted in: Treatments & Research

We received this update today in response to our questions around progress towards access in the UK.

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Invitation to Join Study: Experiences of Living With, or Caring for Someone who has, SMA

21 May 2020 / Posted in: Information, Support

A healthcare market research company working for a research client and pharmaceutical company wants to understand patient and caregiver journeys and any treatment routines. Participants will be paid £35 for each completed survey, with 10% donated to one of the company's registered charities. Registrations of interest close 29th May.

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AveXis receives European Commission (EC) approval for the gene therapy Zolgensma®

19 May 2020 / Posted in: Treatments & Research

The EC has granted approval for Zolgensma® for the treatment of babies and young children who have 5q SMA with a bi-allelic mutation in the SMN1 gene and either a clinical diagnosis of SMA Type 1 or up to three copies of the SMN2 gene.

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NICE & NHS England Answer Nusinersen FAQs on MAA and Access for Children

19 May 2020 / Posted in: Treatments & Research

We finally have answers to some of the questions the patient groups asked NHS England and NICE in August 2019. Other FAQs posed were about access for adults, data collection and the treatment itself. These should be coming soon.

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Do you live with a neuromuscular condition and experience swallowing difficulties, or care for someone who does?

18 May 2020 / Posted in: Information, Support

If so, please consider sharing your experiences by completing an online survey led by a specialist team at The National Hospital for Neurology and University College London. Survey closes 26th July.

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PhD Scholarship Funding Cancelled This Year

18 May 2020 / Posted in: Treatments & Research

Due to the financial effect COVID-19 is having on Muscular Dystrophy UK’s ability to generate income, they have with great regret had to cancel their 2020 Grant Round. This includes the PhD scholarship scheme we were undertaking jointly with them. Sadly due to the impact on our funding as well, it would be unrealistic for SMA UK to organise our own process.

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