Further Positive Results in NURTURE Study with Pre-Symptomatic SMA Patients Treated with Nusinersen

12 June 2020 / Posted in: Treatments & Research

New results from the NURTURE study show that pre-symptomatic SMA patients treated with nusinersen continue to demonstrate sustained benefit from treatment.

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NICE / NHS England Answer the Patient Groups FAQS about Adults’ Access to Nusinersen Treatment in England

11 June 2020 / Posted in: Treatments & Research

NHS England and NICE have provided further responses to these FAQs, around adults' access to nusinersen, raised in August 2019 by the patient groups.

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Information Statement from the NHS England Clinical Panel for Nusinersen

11 June 2020 / Posted in: Treatments & Research

This is about access to the risdiplam Compassionate Use Programme for anyone who has SMA Type 1 or 2 who is already receiving nusinersen but who has had their treatment interrupted because of COVID-19.

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Impact of COVID-19 on SMA UK: June Update

10 June 2020 / Posted in: Information

Read the latest about staff furloughing and what else is going on at SMA UK at the moment.

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NICE & NHS England Answer Further FAQS about the Nusinersen MAA; Biogen Gives Update on Global Access

09 June 2020 / Posted in: Treatments & Research

You can read these communications in this update. Answers to our FAQs about adult services will also be coming soon.

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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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