Latest News: Treatments & Research

SMA UK and Many Others Respond to NICE’s Consultation on Access to Risdiplam

23 June 2021 / Posted in: Treatments & Research

Our thanks to the many people we talked with who planned to make their own personal responses. These will powerfully echo the voices of those who responded to our surveys and spoke up at the committee meeting. Together, we are calling for access for all for whom risdiplam is a clinically safe option.

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Evrysdi™ Improves Motor Function in Pre-Symptomatic Babies

14 June 2021 / Posted in: Treatments & Research

In response to a request from SMA Europe, Roche has shared new interim data from two studies evaluating risdiplam in people with SMA. The trials included pre-symptomatic babies (RAINBOWFISH) or patients who have been previously treated with other SMA-targeting therapies (JEWELFISH). The findings were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting.

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NICE publishes its Final Evaluation Document on Zolgensma

04 June 2021 / Posted in: Treatments & Research

Today, NICE confirmed its recommendations originally announced on 8th March 2021. There is now an appeal period closing on 18th June. Final publication is scheduled for 7th July 2021.

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How Scales and Measurements Will Work Now for England’s MAA for Nusinersen

04 June 2021 / Posted in: Treatments & Research

The nusinersen MAA requires data to be collected about each individual person being treated. This is so that you and your clinical team can monitor if the treatment is working for you / your child – bearing in mind that stabilisation of your / your child’s condition is a key aim. Here we explain how the scales and measurements will work going forwards.

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Evidence and Summary that led to NICE’s Type 3 Access to Nusinersen Decision Published

04 June 2021 / Posted in: Treatments & Research

Today you can read what sat behind this momentous decision first announced on 4th May. Over almost two years, SMA UK worked hard to advocate and contribute to this, amplifying the voices of the families and adults who have been impacted so distressingly by their exclusion from treatment.

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Update from the National Multidisciplinary Team (NMDT) on the progress of the Zolgensma Treatment Programme in England

03 June 2021 / Posted in: Treatments & Research

This newly formed team of lead consultants from the four infusion centres is meeting very regularly. They are working on the many operational aspects of this new national programme that need to be in place to ensure that it is delivered in the best most equitable way possible.

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NICE Asks for Comments on its Appraisal Consultation Document (ACD) for Risdiplam

02 June 2021 / Posted in: Treatments & Research

NICE’s initial position is to say 'no' to approving risdiplam for NHS funding. This is disappointing news, but not unusual at this stage, and NICE is asking for comments on this by 23rd June, which they will review at their next committee meeting on 13th July. We will be responding to the consultation paper, again advocating for access for all as soon as possible. We remain hopeful that this process is similar to other appraisals whereby once more work is completed, the initial 'no' changes to ‘yes’.

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First Baby in England Treated with Zolgensma

01 June 2021 / Posted in: Treatments & Research

It’s very heartening to see the first child with SMA Type 1 being treated with this ground-breaking gene therapy via the NHS at Evelina Hospital. As we support families through this understandably anxious period, we are doing all we can to ensure that those currently waiting will, as soon as possible, be able to have full discussions with their clinical team to see if this treatment option will also be appropriate for their child.

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Invitation to Parliamentary Meeting on Newborn Screening

28 May 2021 / Posted in: Information, Treatments & Research

On 9th June, the APPG on Rare, Genetic and Undiagnosed Conditions will be holding a meeting on newborn screening. The Newborn Screening Alliance is working with Genetic Alliance UK to improve access to newborn screening. You can ask your MP to attend: appg@geneticalliance.org.uk

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Access to Zolgensma – Latest Updates on the Q&As for England

26 May 2021 / Posted in: Treatments & Research

We have received some further answers from NHS England for our Q&As. We will continue to update these as soon as we receive new information, giving answers to the many questions people have asked.

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