New Survey Launched to Understand Activity Needs of Disabled Children

16 January 2020 / Posted in: Information, Support

The ukactive Research Institute has announced a three-year research project with national disability charity, Sense, to understand better the needs of parents and families of disabled children. Survey closes 15th March.

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Novartis Provided an Update on its Phase 1/2 Study in SMA Type 1

15 January 2020 / Posted in: Treatments & Research

Like Roche’s risdiplam (RG7916), branaplam (LMI070) is a small molecule drug that increases the amount of SMN protein made by the SMN2 gene. The study is progressing well, with a total of 29 infants receiving the study treatment.

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Scholar Rock Summarises Progress To Date With SRK-015

14 January 2020 / Posted in: Treatments & Research

SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients. Here, Scholar Rock summarise the latest progress.

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Roche Announces Global Compassionate Use Programme for Risdiplam

13 January 2020 / Posted in: Treatments & Research

This very positive development enables those who have SMA Type 1 global access to this promising treatment, with plans to expand access to those with SMA Type 2 later in the year. Roche UK has issued a statement with all the relevant information for UK patients.

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Do you or someone in your family have SMA Type 3? Are you / they currently not eligible for nusinersen treatment via the NHS?

10 January 2020 / Posted in: Treatments & Research

Complete our survey(s) by 16th February to let us and the other patient groups (MDUK and TreatSMA) know what impact this has had on you / your family.

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From April 2020: Free Hospital Parking for Some Patients, Staff and Carers

10 January 2020 / Posted in: Information, Support

Disabled people, frequent outpatient attenders, parents of sick children staying overnight and staff working night shifts, will not have to pay for NHS car parking from April 2020.

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NICE Committee Meeting to Review the Possibility of Recommending that Access to the Treatment Zolgensma is Funded by the NHS Delayed

06 January 2020 / Posted in: Treatments & Research

NICE advised on 23rd Dec that the first committee discussion scheduled to take place on Thursday 26 March will not now take place. The evaluation will be rescheduled into the work programme and the updated timelines will be communicated, once known.

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SMA Europe Sets Out its Concerns about the AVXS-101 Zolgensma Global Compassionate Use Programme Announced in December 2019

06 January 2020 / Posted in: Treatments & Research

As members of SMA Europe, we endorse this statement made by SMA Europe on 26th December. It outlines the many concerns there are about this programme.

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Petition On Social Care Reform

20 December 2019 / Posted in: Information, Support

The Neurological Alliance has launched a joint campaign, with Change.org and Epilepsy Action, to raise the profile of people with neurological conditions in the conversation around social care. Find out more and sign the petition.

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Limited Global Access to Zolgensma announced by Avexis

20 December 2019 / Posted in: Treatments & Research

Clinicians and SMA patient groups in SMA Europe have welcomed the news that Avexis will be launching a global compassionate use programme in January 2020 for eligible children under 2 years of age, but have concerns about how it will operate.

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