Latest News: Treatments & Research

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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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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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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Physiotherapy - Based Measures (HINE, CHOP INTEND, HMFSE) for People with SMA and how they are used to Monitor Treatment Outcomes

02 September 2019 / Posted in: Treatments & Research

In this new article, our Clinical Care Research Correspondent, Dr Alex Murphy, summarises: the way some of the more commonly used physiotherapy-based outcome measures in SMA work; the research that shows how these measures would be expected to progress for those with SMA who have not accessed drug treatments.

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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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