Survey on Accessible Transport and Parking

01 July 2019 / Posted in: Information, Support

Business Disability Forum has launched this survey seeking the views of disabled people or those who have a long-term health condition, on using public transport and accessible parking, in the UK. Survey closes 8am on Wednesday 10th July.

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Sign Open Letter Calling On NHS England To Keep Its Promise To Protect The Children’s Hospice Grant

01 July 2019 / Posted in: Information, Support

Together for Short Lives has found that NHS and local council cuts are hitting lifeline care for seriously ill children. Gabriella Walker, a bereaved parent, is inviting children’s palliative care services, families of children with life-limiting conditions, and the general public to add their name to her letter to the Health and Social Care Secretary, calling for urgent action.

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Guide to the 2017 International Standards Of Care For SMA Launched Today

26 June 2019 / Posted in: Treatments & Research

The SoC is now available via the TREAT-NMD website. It describes what assessments and interventions families and adults should expect to find in any neuromuscular centre anywhere.

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Your Views on the Possible Provision of Zolgensma by NHS England, for Infants who have SMA Type 1

25 June 2019 / Posted in: Treatments & Research

Patient groups would like to find out what you think about this new treatment so that we can include your views in our submissions to the National Institute for Health and Care Excellence (NICE). Our survey closes on 21st July 2019.

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Update re Nusinersen MAA

20 June 2019 / Posted in: Treatments & Research

We are so sorry to hear of the continued heightened distress in the SMA Community caused by NICE / NHS England’s decisions and communications to date. We are doing all we can to obtain a reply to the letter sent by clinicians and the patient groups on 14th June. This was sent following consultation, advice and a joint decision that this step was the most promising way to obtain a change to the MAA.

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Children’s Hospice Week: 17th – 23rd June

14 June 2019 / Posted in: Information, Support

This is the only week in the year dedicated to raising awareness of children’s hospice and palliative care services across the UK, and those they support. This year’s theme is ‘Moments that Matter’.

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Patient Advocacy Groups and Clinicians write to NICE & NHSE about the nusinersen MAA and the impact this has had on the SMA Community

14 June 2019 / Posted in: Treatments & Research

Today we have co-signed two letters to address the concerns that are being widely expressed about the nusinersen Managed Access Agreement (MAA).

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Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers

11 June 2019 / Posted in: Treatments & Research

Scholar Rock announced that SRK-015 was well-tolerated in healthy volunteers across all tested doses and shows robust and durable target engagement, supporting evaluation in the Phase 2 trial in SMA, which is currently ongoing.

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NICE Announces Appraisal of the Treatment Zolgensma for Infants with SMA Type 1

10 June 2019 / Posted in: Treatments & Research

Following an application by AveXis, NICE is progressing an appraisal of this treatment to see if it will recommend it for provision by NHS England for the treatment of infants with SMA Type 1. We will be asking the Community for their views and making a submission by the deadline of 31st July.

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Access to Nusinersen in England Update

10 June 2019 / Posted in: Treatments & Research

We were extremely concerned to read some of the eligibility criteria in the draft MAA. We raised these concerns immediately with NICE, NHS England and Biogen. We continue to work with MDUK, Treat SMA and clinicians to establish the next most constructive steps at what is a most delicate stage in this long, difficult and distressing process. This needs very careful thought. We will communicate further on this as soon as possible this week.

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