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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Roche Presents Data From The Trials At Annual Congress Of The World Muscle Society

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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Quicksilver Media Looking For Families Accessing Spinraza To Take Part In Documentary

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

Quicksilver media are currently making a documentary about the possible implications for drug budgets of any UK-US trade deal, for Channel 4's Dispatches strand, and are looking for families with children who have recently (within the last 4 months) started treatment with Spinraza / nusinersen.

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All Party Parliamentary Group on Access to Medicines and Medical Devices makes recommendations for the NICE Methods Review

25 September 2019 / Posted in: Treatments & Research

Representing the views of a broadly-based group of stakeholders (including SMAUK) this report will be taken to the Government, the NHS, and NICE, to continue dialogue about what should change to support better patient access to innovative therapies.

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Biogen Community Update on DEVOTE Clinical Trial

20 September 2019 / Posted in: Treatments & Research

Announced publicly at the 13th Congress of the European Paediatric Neurology Society (EPNS), the DEVOTE study will evaluate if higher dosing of nusinersen can provide improved efficacy in the treatment of SMA across a broad population of patients.

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SMA Matters Autumn 2019

16 September 2019 / Posted in: Information, Support

This edition, and previous editions, of our SMA Matters newsletter are now available to download from our website.

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Survey About Your Child’s Experience Of Sport & Activity

12 September 2019 / Posted in: Information, Support

Activity Alliance, a national charity, wants to hear from parents and disabled children about their experience of sport and activity.

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