#GiveMeABreak Campaign - Together for Short Lives

07 October 2020 / Posted in: Information, Support

Together for Short Lives is asking families in England to write to their local MP to ask them to contact Chancellor Rishi Sunak about funding short breaks. This follows a report which shows the positive impact that short breaks have for families.

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“Think 3” Awareness Campaign Launched Today for Parents and Carers

06 October 2020 / Posted in: Information, Support

This campaign aims to raise awareness of three motor developmental milestones that babies are expected to reach at up to 3 months of age so that those not reaching them are identified and referred quickly; this could be signs of SMA or another neuromuscular condition.

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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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