NICE Committee Meeting to Review the Possibility of Recommending that Access to the Treatment Zolgensma is Funded by the NHS Delayed

06 January 2020 / Posted in: Treatments & Research

NICE advised on 23rd Dec that the first committee discussion scheduled to take place on Thursday 26 March will not now take place. The evaluation will be rescheduled into the work programme and the updated timelines will be communicated, once known.

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SMA Europe Sets Out its Concerns about the AVXS-101 Zolgensma Global Compassionate Use Programme Announced in December 2019

06 January 2020 / Posted in: Treatments & Research

As members of SMA Europe, we endorse this statement made by SMA Europe on 26th December. It outlines the many concerns there are about this programme.

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Petition On Social Care Reform

20 December 2019 / Posted in: Information, Support

The Neurological Alliance has launched a joint campaign, with Change.org and Epilepsy Action, to raise the profile of people with neurological conditions in the conversation around social care. Find out more and sign the petition.

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Limited Global Access to Zolgensma announced by Avexis

20 December 2019 / Posted in: Treatments & Research

Clinicians and SMA patient groups in SMA Europe have welcomed the news that Avexis will be launching a global compassionate use programme in January 2020 for eligible children under 2 years of age, but have concerns about how it will operate.

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Clinicians & Patient Groups Requesting AveXis Clarification About Potential Limited Global Access to Zolgensma

19 December 2019 / Posted in: Treatments & Research

We're aware that complex and sensitive information regarding potential but very limited global access to the gene therapy treatment Zolgensma has been shared online. Clinicians and patient groups have expressed their concerns to the drug company AveXis requesting, as a matter of urgency, public clarification of the position.

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New SMA Voices: Gary Shares His Experiences

18 December 2019 / Posted in: Information, Support

In this video, Gary looks back at his childhood and forward to present day, and shares his experiences of what it’s like to live with Spinal Muscular Atrophy.

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Information and Support over the Christmas Break

11 December 2019 / Posted in: Information, Support

Our office is closed from 1pm on Friday 20th December 2019. We re-open on Thursday 2nd January 2020 at 9.00am. Here are links to where you can access support and information about SMA during this time.

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Together for Short Lives Launches Youth-Led Creative Artwork Project

10 December 2019 / Posted in: Information, Support

Called 'This Is Me', the project provides an opportunity for young people with life-limiting conditions in the UK to express themselves through the creative arts. Submissions deadline is 20th January 2020.

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Support the #NeuroPledge2019

03 December 2019 / Posted in: Information, Support

The Neurological Alliance are asking parliamentary candidates to take the #NeuroPledge2019. It commits them to campaigning for improvements for people with neurological conditions, if they become an MP. Find out how you can get involved and write to your prospective MP.

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Nusinersen Managed Access Agreement – Update on Progress with Services for Adults

02 December 2019 / Posted in: Treatments & Research

NHS England has let the Patient Advocacy Groups (PAGs) know which hospitals now have contracts to offer adults treatment, and their plans to address any service gaps.

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