Independent Real-World Study Demonstrates Significant Improvements for Adults who have SMA Type 3, Treated with Nusinersen

06 October 2020 / Posted in: Treatments & Research

In response to a request from SMA Europe, Biogen has sent this community update which includes the observation that the most relevant treatment effect was seen in those with SMA Type 3 who were unable to walk without assistance at the start of treatment.

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Roche: Update on Part 1 of the FIREFISH Risdiplam Clinical Trial

02 October 2020 / Posted in: Treatments & Research

Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. The data were presented at the virtual 25th International Annual Congress of the World Muscle Society.

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Disabled Children and Young People Entitled to Free Flu Jab this Winter

30 September 2020 / Posted in: Information, Support

Winter is coming, and at-risk groups entitled to a free flu vaccination this year include children, carers, people with learning disabilities and adults with long-term health and medical conditions.

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Community Update on AVXS-101 Intrathecal Clinical Development Program

29 September 2020 / Posted in: Treatments & Research

Novartis has provided this update regarding their STRONG trial of intrathecal delivery of Zolgensma in older SMA patients.

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Summary of the Outcomes of the Clinical Trials of Risdiplam: September 2020

28 September 2020 / Posted in: Treatments & Research

Our Scientific Research Correspondent, Dr. James Sleigh, has compiled this summary of the four trials of risdiplam: FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH.

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Should NICE recommend that the NHS funds risdiplam and, if so, who for? Let us know your views

25 September 2020 / Posted in: Treatments & Research

Based on the clinical and economic data from the manufacturer (Roche), NICE will be considering the clinical and cost effectiveness of risdiplam, and the views of patients and clinicians. We want to be sure to pass on your views to NICE when we make our Patient Group submission in October. Deadline: 11th October 2020.

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Roche Answers Patient Group Questions about the UK Early Access to Medicines Scheme (EAMS) for Risdiplam

23 September 2020 / Posted in: Treatments & Research

This information is provided in response to a series of questions shared jointly with Roche in writing by UK patient organisations SMA UK, Muscular Dystrophy UK, and TreatSMA.

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Baby Loss Awareness Week: 9th-15th October

23 September 2020 / Posted in: Information, Support

This year, the theme is highlighting the isolation many people experience after pregnancy and baby loss – women, partners, other family members and friends. The effect of social distancing from Covid-19 has had a major impact on access to care and support and has complicated grief and responses to pregnancy and baby loss.

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DCP - Back to School Survey

21 September 2020 / Posted in: Information, Support

Following the success of the Disabled Children's Partnership's survey in the spring about being #LeftinLockdown, they have launched this new survey to gather information on how the return to school has gone for disabled children. Deadline to take part is 5th October.

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COVID-19 - Survey on Flying with a Disability

21 September 2020 / Posted in: Information, Support

Josh Wintersgill, who has SMA, is the founder and director of easyTravelseat. They are running a survey to gather views around how disabled people feel about flying in the current climate, and to find out more about the impact that Covid-19 has had.

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