Patient and Caregiver Survey: Understanding Your Views on Spinal Muscular Atrophy Treatments

19 August 2019 / Posted in: Information, Treatments & Research

This survey by Acaster Lloyd Consulting Ltd, is designed to understand your views about the importance of different aspects of SMA treatments and any trade-offs you would consider making. The study findings will be used to help healthcare decision-makers (e.g. NICE in the UK) understand how important different aspects of SMA treatments are to adults and caregivers of children with SMA Types 2 and 3.

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Do You Know Someone Who Deserves or Needs a Brand-New Garden Makeover?

13 August 2019 / Posted in: Information

ITV1's garden makeover show 'Love Your Garden with Alan Titchmarsh' is back in Autumn 2019. They're looking for families / individuals to apply or nominate someone who could benefit. Closing date 30th September.

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Access to Zolgensma Survey and Submission Sent to NICE

08 August 2019 / Posted in: Treatments & Research

Thanks to all who responded to our survey asking for your views on access to Zolgensma for infants with SMA Type 1. We've passed them on to MDUK & TreatSMA and used them in our joint submission with MDUK. Click here to see the results and our submission to NICE.

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Biogen’s Community Statement Answers Some Questions

07 August 2019 / Posted in: Treatments & Research

SMA UK, MDUK and TreatSMA jointly asked Biogen a number of questions relating to access to the nusinersen Managed Access Agreement with NHS England, and access in the UK in general.

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Potential SMA Treatment Granted Orphan Medicinal Product Designation By The European Medicines Agency (EMA)

31 July 2019 / Posted in: Treatments & Research

Cytokinetics announced that EMA has granted Orphan Medicinal Product Designation to reldesemtiv for the potential treatment of SMA. Reldesemtiv is a drug that has been shown, in the laboratory, to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue.

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NICE Publishes Final Guidance Following Its Appraisal Of Nusinersen

24 July 2019 / Posted in: Treatments & Research

As there have been no appeals, access to the treatment will be as outlined in the Managed Access Agreement. We have not forgotten those who don't currently have access and will continue to work to change this.

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Registration for SMA Europe’s 2nd International Scientific & Clinical Congress on SMA Now Open

19 July 2019 / Posted in: Treatments & Research

The conference will be held at Génocentre in Evry, south of Paris between 5th & 7th February 2020 and is open for healthcare professionals and researchers in SMA to attend. The congress will feature a series of talks and workshops covering key scientific and clinical research fields in SMA.

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NCB Survey: Experiences Of Using SEND Information, Advice and Support Services

18 July 2019 / Posted in: Information, Support

The National Children’s Bureau are carrying out a survey about people’s experience of using SEND information, advice and support services. Your answers to the questions will help the Government understand how good the services are.

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Cardiff Airport Invites Passengers To Join PRM & Disability Forum

11 July 2019 / Posted in: Information

On 4th September, Cardiff Airport is holding a meeting to explore ideas, and to agree the scope for their proposed Passengers with Reduced Mobility (PRM) and Disability Forum. Members of the public and third sector organisations are invited to attend.

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Contact's Resources For A Stress-Free Summer

11 July 2019 / Posted in: Information, Support

The summer holidays are very nearly upon us and Contact for Families with Disabled Children have put together their top tips for making summer as stress-free and fun as possible – this includes a link to their guide “Holidays, Play and Leisure”.

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