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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Our Privacy Policy Has Been Updated

29 April 2021 / Posted in: Information

Following the UK’s exit from the E.U on the 1st January 2021, we have updated our Privacy Policy to reflect the changes to the GDPR (General Data Protection Act).

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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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Spinal Muscular Atrophy UK Appoints New CEO

23 April 2021 / Posted in: Information

SMA UK is pleased to announce that Angela Smith-Morgan will be joining the charity as CEO from 26th April.

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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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Sense Campaign - Left Out Of Life

22 April 2021 / Posted in: Information, Support

Sense have launched this campaign, which is about taking action to tackle loneliness among disabled people and making society more inclusive. The campaign is calling for more investment in services to tackle loneliness and offer mental health support for disabled people. Sign their pledge and play your part in making society more inclusive.

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BBC News Survey - How Covid-19 has Affected Deaf and Disabled People

21 April 2021 / Posted in: Information, Support

BBC News is carrying out a survey to understand how Covid-19 has affected deaf and disabled people. They are trying to reach as many people as possible to get the biggest, most detailed picture they can.

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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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