Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers

11 June 2019 / Posted in: Treatments & Research

Scholar Rock announced that SRK-015 was well-tolerated in healthy volunteers across all tested doses and shows robust and durable target engagement, supporting evaluation in the Phase 2 trial in SMA, which is currently ongoing.

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NICE Announces Appraisal of the Treatment Zolgensma for Infants with SMA Type 1

10 June 2019 / Posted in: Treatments & Research

Following an application by AveXis, NICE is progressing an appraisal of this treatment to see if it will recommend it for provision by NHS England for the treatment of infants with SMA Type 1. We will be asking the Community for their views and making a submission by the deadline of 31st July.

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Access to Nusinersen in England Update

10 June 2019 / Posted in: Treatments & Research

We were extremely concerned to read some of the eligibility criteria in the draft MAA. We raised these concerns immediately with NICE, NHS England and Biogen. We continue to work with MDUK, Treat SMA and clinicians to establish the next most constructive steps at what is a most delicate stage in this long, difficult and distressing process. This needs very careful thought. We will communicate further on this as soon as possible this week.

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'Give it Back' Campaign launched by DCP

03 June 2019 / Posted in: Information, Support

Today sees the launch of 'Give it Back' - a new campaign from the Disabled Children's Partnership to encourage the government to fill the £434million gap in social care funding for disabled children in England through the upcoming Spending Review.

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NICE Releases Nusinersen Final Appraisal Determination (FAD), Draft MAA, and Committee Papers

03 June 2019 / Posted in: Treatments & Research

The FAD will form the guidance to the NHS and the committee papers summarise the information it considered in the process of getting to this stage. We note that the draft MAA still requires some further clarity and development. Jointly with MDUK and TreatSMA we have already raised concerns around this and NICE and NHS England have committed to respond to these at the earliest possible time.

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SANDS Awareness Month - Finding Your Way

03 June 2019 / Posted in: Information, Support

June is SANDS awareness month. 2019's theme is ‘Finding Your Way’ after pregnancy and baby loss and seeking support in a way that is right for anyone needing it, with a particular focus on support for men and helping them find a way through grief. Find out more here including how to support the campaign.

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New Q&A - Prof. Laurent Servais

31 May 2019 / Posted in: Treatments & Research

For our latest Q & A we have Laurent Servais, a clinical researcher recently appointed Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre.

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Roche presented data from its risdiplam pivotal FIREFISH and SUNFISH studies at the 2019 American Academy of Neurology annual conference

31 May 2019 / Posted in: Treatments & Research

Risdiplam is an orally administered small molecule which encourages the SMN 2 “back-up gene” to produce functional SMN protein.

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Survey Reviewing the Supply and Use of Wheelchairs from the NHS

29 May 2019 / Posted in: Information, Support

This survey is designed to capture information on service user experience in the Greater Manchester area of accessing and using a wheelchair (manual, battery powered or electric) over the last 5 years, and to identify both user and carer priorities. Survey closes: 28th June.

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Childhood Project Survey

29 May 2019 / Posted in: Information, Support

The NCB and British Academy want to find out what children and young people aged 11 to 18 think and feel about Childhood. They want to find out how the decisions that governments make affect children and young people’s lives as they are growing up.

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