Latest News: Treatments & Research

Zolgensma Infusion Centres Announced

14 May 2021 / Posted in: Treatments & Research

We’re pleased that NHS England and NHS Improvement have shared this today to keep the Community up to date with developments. Click through to see the update and list of centres.

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Which Centres are Providing Nusinersen and / or Risdiplam?

12 May 2021 / Posted in: Treatments & Research

Following further enquiries, we have summarised the information from NHS England’s 10th May notice about this in a table that may be easier to read. You will need to check what your Centre’s position is in terms of the guidelines that have been issued.

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When will we know if the NHS will fund Risdiplam?

11 May 2021 / Posted in: Treatments & Research

Today, 3 patient experts will be speaking up for risdiplam, for all for whom it is clinically safe, at the first NICE committee meeting considering whether to recommend the treatment for NHS funding. We’ve outlined the stages and hoped-for timeline for when we’ll know the outcome.

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New Guidelines To Improve Access To Nusinersen At Centres

10 May 2021 / Posted in: Treatments & Research

NHS England has just released guidelines about which children / adults should now be prioritised at centres actively providing nusinersen treatment, and what Centres should do if they’re having delays in starting treatment.

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Safety Signal for Zolgensma and Label Changes

05 May 2021 / Posted in: Treatments & Research

Novartis Gene Therapies have issued a community update which has been forwarded to all members of SMA Europe. All clinicians who will be discussing Zolgensma as a possible treatment option for a child will be aware of this update and be able to discuss what this means for them.

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NICE Agrees to Access to Nusinersen for Those who have SMA Type 3 and Aren’t Able to Walk

04 May 2021 / Posted in: Treatments & Research

SMA UK is delighted with this decision which comes after almost two years of advocacy and hard work by families and adults - who have been impacted so distressingly by their exclusion from treatment - their clinicians, patient groups and Biogen. This decision also removes the rule which has meant that those who lose the ability to walk after 12 months of treatment or children who do not regain this ability, wouldn’t be eligible for it to continue.

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Webinar Recording - 'Zolgensma Treatment for SMA Type 1 in the UK'

29 April 2021 / Posted in: Treatments & Research

Our thanks to the leading health professionals who answered questions from the community and led this informative webinar, recorded 21st April 2021, about the treatment and what is going on to progress access. Watch the recording, read some of the key points, and see the answers we have so far to the many questions people have asked.

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New Global Clinical Trial for Onasemnogene Abeparvovec (Zolgensma) Announced

23 April 2021 / Posted in: Treatments & Research

SMA UK welcomes a new global clinical trial that will be investigating the safety, tolerability and efficacy of intravenous Zolgensma in children who have SMA and weigh between 8.5 kgs and 21 kgs.

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Biogen Presents New Data from its DEVOTE Study of Spinraza (nusinersen)

22 April 2021 / Posted in: Treatments & Research

Biogen presents new data from its DEVOTE study of Spinraza (nusinersen) as well as an update on biomarkers and digital tools to enhance disease monitoring, at the 73rd American Academy of Neurology (AAN) Annual Meeting.

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Evrysdi Continues to Improve Motor Function and Survival in Babies with SMA Type 1

19 April 2021 / Posted in: Treatments & Research

Roche has shared new two-year data from part 2 of its FIREFISH study. FIREFISH is assessing Evrysdi™ (risdiplam) in the treatment of infants aged 1-7 months at enrollment, with symptomatic SMA Type 1. The data build upon one-year findings from FIREFISH Part 2, which measures the ability to sit without support for at least five seconds as its primary endpoint.

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