Thank You and Welcome – Changes to our Trust Board

28 January 2021 / Posted in: Information

Heartfelt thanks for all he has done to our long-serving Trustee Hugo Van Vredenburch, who steps down next month but remains committed to supporting our fundraising efforts. We are delighted to welcome Dr. Anne-Marie Childs - Consultant Paediatric Neurologist - as a new Trustee.

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Treatment Decisions in Newly Diagnosed Families with SMA

27 January 2021 / Posted in: Treatments & Research

A group of patient representatives from SMA Europe responded to European neuromuscular experts’ consensus statements on gene replacement therapy for SMA. Their short article, where they highlight some of the considerations that must be taken into account when considering treatment, was published in the European Journal of Paediatric Neurology.

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MDUK - Your Experience of the COVID-19 Pandemic

27 January 2021 / Posted in: Information, Support

Muscular Dystrophy UK are running a survey with the opportunity for you to share your experiences of coping with the pandemic and accessing services. They are keen to learn about any positive changes you have experienced in the way services have been delivered, as well as exploring the challenges you have faced.

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Work and Pensions Committee Inquiry into the Disability Employment Gap

27 January 2021 / Posted in: Information, Support

Thank you to the young adult network members who worked with us in December to submit a response to the questions raised. We also drew on the experiences others have relayed to our Support Services Team.

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National Strategy for Disabled People: Survey Launched

22 January 2021 / Posted in: Information, Support

The public survey is a chance for disabled people and their carers, friends and family, to share views and life experiences. The responses will help the government understand the barriers faced by disabled people and what it can do to improve disabled people's lives.

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NCB - Living Assessments Recruitment

15 January 2021 / Posted in: Information, Support

Are you a young person or parent with experience of having had a social care assessment? Living Assessments is a five-year project from the National Children's Bureau looking at the decision-making process for providing social care support to children and families, the impact of that support, and the lived experiences of assessments.

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Adept Field Solutions - Research Project

15 January 2021 / Posted in: Information, Support

Market researchers Adept Field Solutions are running a project, sponsored by a pharmaceutical company, seeking the views of people in the UK who are living with SMA and their families / partners / caregivers. Those who take part will receive £50.

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First patient treated in Biogen’s global RESPOND study of nusinersen, following treatment with Zolgensma™

11 January 2021 / Posted in: Treatments & Research

The Phase 4 open-label, multicentre study will examine the clinical benefit and safety of nusinersen treatment in infants and children with SMA who have unmet clinical needs following previous treatment with Zolgensma.

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AMRC - Write to Boris Johnson to Save Charity-Funded Medical Research

11 January 2021 / Posted in: Information, Treatments & Research, Support

AMRC has reported on the devastating impact of covid on medical research charities who have had to make drastic cuts to their research funding. You can sign AMRC's letter calling for the Government to provide vital funding support to protect medical research and patients.

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New UK Rare Diseases Framework Published January 2021

10 January 2021 / Posted in: Information, Treatments & Research

The Framework demonstrates the commitment of UK governments to improve the lives of people with rare conditions, and address key challenges, over the next five years.

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