Action For Access

25 October 2019 / Posted in: Information, Treatments & Research, Support

Genetic Alliance UK have launched this campaign to improve access to potentially life-changing treatments for people affected by rare diseases (including SMA). We're backing their campaign and calling for #ActionForAccess. Find out how you can get involved too.

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Two new studies show positive effects of nusinersen in later-onset SMA

24 October 2019 / Posted in: Treatments & Research

Two small, observational studies of the effects of nusinersen in patients with later-onset SMA have recently been published, both of which describe clinically meaningful improvements caused by the treatment.

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Biogen Communicates Updated NURTURE Study Results for the European Community

24 October 2019 / Posted in: Treatments & Research

The following updated results were formally presented at the Cure SMA Annual SMA Conference in Anaheim, California and the 5th Congress of the European Academy of Neurology (EAN) in Oslo, Norway.

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SMA Europe Report from Warsaw, Poland

24 October 2019 / Posted in: Treatments & Research

Polish member organisation, Fundacja SMA, were excellent hosts for SMA Europe’s biannual meeting between 10th & 12th October. The General Assembly is made-up of Patient Organisations from 19 member countries with up to two delegates from each (for SMA UK - Liz Ryburn, Support Services Manager, and Hassan Sobati, Trustee).

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Interim Data from the STRONG Trial Indicate that Zolgensma Treatment Improves Motor Function in Older SMA Type 2 Patients

10 October 2019 / Posted in: Treatments & Research

The data show an increase in motor function scores among older SMA Type 2 patients following a one-time intrathecal (IT) administration of the treatment.

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Three ‘Pillars’ to Deliver Better Support and Care for Disabled Children and Their Families

10 October 2019 / Posted in: Information, Support

The Disabled Children's Partnership, of which we are members, have identified three pillars to underpin and ensure improved support for disabled children and their families. Read more in this blog from Stephen Kingdom, DCP Campaign Manager.

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Children who have SMA Wanted for Interview about New Product Development

10 October 2019 / Posted in: Information, Support

Healthcare Fieldwork's client are looking to interview children who have SMA and who are aged between 5-15 years old (and parents / carers) at their offices in Cambridge. Participants will receive £120 as a thank you for taking part.

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Patients & Public Wanted To Help With Research About Swallowing Disorders In Neuromuscular Conditions

08 October 2019 / Posted in: Information, Treatments & Research

The purpose of this study is to help find out what the priorities are for people with a neuromuscular condition who have swallowing difficulties, and the experiences of family members and carers. The first meeting has been set for 25th October so if you'd like to register your interest, please do so as soon as possible.

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PTC Therapeutics Press Release - Risdiplam (RG7916)

07 October 2019 / Posted in: Treatments & Research

Data presented at the Annual Congress of the World Muscle Society, demonstrated the ongoing benefit of risdiplam (RG7916) for the treatment of all Types of SMA. Risdiplam is a small molecule drug taken orally, which targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of functional SMN protein.

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Biogen’s Community Update Concerning Those Transferring from the Nusinersen EAP to the Managed Access Agreement

27 September 2019 / Posted in: Treatments & Research

Some families whose children are transferring from the Expanded Access Programme to the MAA have expressed concerns about future treatment. We contacted Biogen and NHS England for answers and reassurance.

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