International Research Survey: Coronavirus

14 May 2020 / Posted in: Information, Support

A collaboration of international researchers, including researchers from University College London, are interested in how the current coronavirus pandemic is affecting disabled people and their families.

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Scholar Rock Reports Delays in Interim Trial Results Due to COVID-19

13 May 2020 / Posted in: Treatments & Research

Scholar Rock reported on the recent progress and upcoming milestones for the TOPAZ Phase 2 clinical trial of SRK-015 in patients who have SMA Type 2 or 3, in the light of the COVID-19 pandemic.

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Research Survey to Understand the Impact of COVID-19 on People who have a Rare Disease, and their Caregivers

12 May 2020 / Posted in: Information, Support

Researchers at Queen’s University Belfast and University of Aberdeen are conducting this online international survey. It's available to complete until the end of May.

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EURODIS Survey to Understand how the COVID-19 Pandemic is Affecting those who have a Rare Disease, and their Carers

12 May 2020 / Posted in: Information, Support

The goal is to voice the specific needs of rare disease patients’ in relation to the COVID-19 pandemic and to propose practical solutions to ensure rare diseases patients are take into account when handling the pandemic.

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Request for NICE to Reconsider the Decision to Suspend the Appraisal Process for risdiplam

07 May 2020 / Posted in: Treatments & Research

Yesterday we sent a letter to NICE, jointly with MDUK and TreatSMA, stating that for the large proportion of the SMA community there is no treatment, and therefore the appraisal of risdiplam is critical, and must continue to be appraised.

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Disabled Children's Partnership Survey: Life in Lockdown

07 May 2020 / Posted in: Information, Support

The Disabled Children's Partnership, of which SMA UK is a member, wants to understand more about the impact of the coronavirus pandemic on disabled children and young people (aged up to 25), their siblings and parents. Deadline to complete it is 9am on Monday 18th May 2020.

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MPs Publish Influential Report on SEND

07 May 2020 / Posted in: Information, Support

The committee concluded that children with SEND are seeing their education, well-being and life chances damaged by failings in SEND provision, and that the Department for Education does not have a grip on mounting pressure in the system.

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Roche UK Confirms Submission of their Application for an Early Access to Medicines Scheme (EAMS)

05 May 2020 / Posted in: Treatments & Research

Roche took this planned step on 4th May. It usually takes approximately 90 days to hear the outcome.

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Roche’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants who have SMA Type 1

28 April 2020 / Posted in: Treatments & Research

Roche has shared an update on part 2 of the FIREFISH clinical trial. FIREFISH Part 2 is a pivotal, global study evaluating risdiplam in 41 infants aged 1 to 7 months old with symptomatic SMA Type 1.

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Disability Rights UK's Petition To Increase Disability Benefits

28 April 2020 / Posted in: Information, Support

In response to the COVID-19 pandemic, the Government announced a temporary emergency increase of £20 a week for both Universal Credit and Working Tax Credits. This petition is calling on the Government to immediately extend this emergency increase to all benefits.

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