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AveXis Issues Community Statement on the SPR1NT Trial

08 May 2018 / Posted in: Research

AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT. They have provided the following community statement.

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Scottish Medicines Consortium (SMC) Decision and Next Steps on Access to Nusinersen

07 May 2018 / Posted in: Research

The SMC has just recommended nusinersen to be funded only for children with SMA Type 1. Whilst we recognise this is a positive step forward, it is hugely disappointing that children, young people and adults with SMA Type 2 or Type 3 who wish to access, and could potentially benefit from, this treatment still don’t have this opportunity. We will now be pushing for a reassessment of the treatment. You can help.

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Biogen Community Update re SMC Routine Funding Recommendation

07 May 2018 / Posted in: Research

Following the Scottish Medicines Consortium's announcement of recommending nusinersen to be funded only for children with SMA Type 1, Biogen has released the following community update.

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Extending Rights to Personal Health Budgets

04 May 2018 / Posted in: Information & Support

This survey seeks views on extending specific groups rights to an integrated personal budget (e.g.people who access wheelchair services whose posture and mobility needs impact their wider health & social care needs) and on the potential to incorporate additional funding streams into that budget.

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Scholar Rock Experimental Drug for SMA Granted Orphan Drug Status

03 May 2018 / Posted in: Research

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SRK-015 for the treatment of SMA. SRK-015 is the lead drug candidate of pharmaceutical company Scholar Rock.

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Novartis Agrees $8.7 Billion Deal to Acquire AveXis

03 May 2018 / Posted in: Research

One of the largest pharmaceutical companies in the world, Novartis, has entered an agreement to purchase the relatively small gene therapy company, AveXis, which has been instrumental to the development and testing of the SMA gene therapy, AVXS-101.

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Access to Psychological Support - Your Views

02 May 2018 / Posted in: Information & Support

Thank you to those of you who completed our surveys. We have forwarded the results and your comments to MDUK so that they can be incorporated into the report they are preparing for the APPG 6-month inquiry. To see the results of the surveys, or if you are seeking emotional support, please click here.

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Survey about Family Finances

24 April 2018 / Posted in: Information & Support

Contact is calling on family members who care for children and young people with additional needs or disabilities to take part in important research about family finances.

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2018 Porsche Muscle Dream Driving Experience Programme 2018 Porsche Muscle Dream Driving Experience Programme

19 April 2018 / Posted in: Our Community

Nominations are now open for the 2018 Porche Muscle Dream Driving Experience Programme! The Muscle Help Foundation is looking to invite 8 young people to join this exciting experience on 18th/19th June at Silverstone.

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Do you have SMA? Are you thinking about having children now or in the future?

12 April 2018 / Posted in: Information & Support

We have a number of resources available here on our website that you may find helpful or of interest, including personal stories that mums with SMA have shared.

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