Latest News: Treatments & Research

Early Access to Medicines Scheme (EAMS) announced for Risdiplam in the UK

17 September 2020 / Posted in: Treatments & Research

Today, the Medicines and Healthcare Products Regulatory Agency (MRHA) announced access to Risdiplam for those who have SMA Type 1 or Type 2, aged 2 months and older, who are not suitable for authorised treatments.

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Biogen: September Community Update on European / Global Access to Nusinersen

14 September 2020 / Posted in: Treatments & Research

Biogen has provided the latest update on access to nusinersen treatment around the world. 29 European countries now have access to nusinersen via regular reimbursement.

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Muscular Dystrophy UK - SMA Seminar

14 September 2020 / Posted in: Information, Treatments & Research, Support

The first seminar of their 'MDUK Muscles Matter 2020' series took place during SMA Awareness Month and was an update on research into SMA and a panel discussion on potential treatments and managing the condition. Watch it here.

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AveXis Renamed Novartis Gene Therapies

03 September 2020 / Posted in: Treatments & Research

Novartis has renamed the previously acquired AveXis - the pharmaceutical company behind the gene therapy treatment, Zolgensma - to Novartis Gene Therapies. The change signifies the growing importance of gene therapy.

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SMA Europe and Partners Launch the European Alliance for Newborn Screening

31 August 2020 / Posted in: Treatments & Research

Early diagnosis and treatment of SMA leads to better outcomes. Launched today, on the last day of SMA Awareness Month, this Alliance brings together all stakeholders (EAMDA, EURORDIS, TREAT-NMD, AveXis, Biogen, and Roche) with the aim, by 2025, for newborn screening programmes in Europe to include a test for SMA.

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Nice Announces Next Stages in Appraisal of Risdiplam / Evrysdi

28 August 2020 / Posted in: Treatments & Research

When we heard in April this year that NICE’s risdiplam appraisal was to be put on hold due to Covid-19, patient groups wrote jointly to NICE urging them to reinstate the process at the earliest possible time due to the great potential this treatment has for the SMA Community. We were pleased to hear this week that the appraisal is again progressing.

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NICE Widens Scope of its Appraisal of Zolgensma

28 August 2020 / Posted in: Treatments & Research

This will now be in line with the European marketing authorisation and will now also include those who have 5qSMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene and, if evidence allows, consideration may be given to a sub-group of people who are pre-symptomatic. This widens the possibilities and is what patient groups have been advocating.

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About the Young Scientists who Make Up the UK SMA Research Conference’s Committee

24 August 2020 / Posted in: Treatments & Research

Six dedicated young PhD students from institutions across the UK are organising an online SMA research conference on 10th & 11th September. Here, you can find out more about them, their work and their interests outside the laboratory!

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FAQs About Roche’s Risdiplam Compassionate Use Programme (CUP) for Those who have SMA Type 1 or 2

21 August 2020 / Posted in: Treatments & Research

These cover who may be eligible for access, the application process and how the treatment programme will work. They also cover some other questions that have been asked about what future access routes to risdiplam there may be for those who have Type 3.

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Biogen: Community Update on European / Global Access to Nusinersen

20 August 2020 / Posted in: Treatments & Research

Biogen has provided the latest update on access to nusinersen treatment around the world. There are now 29 European countries that have access to nusinersen via regular reimbursement.

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