Easter Bank Holiday

08 April 2020 / Posted in: Information, Support

SMA UK staff won’t be working over the Easter Weekend - so will finish at the end of Thursday 9th April and will be back working at 9am on Tuesday 14th. Find out about where to find information, including re COVID-19, during this time.

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Continued Advocacy re: Access to Nusinersen For All Those Who Have SMA Type 3

07 April 2020 / Posted in: Treatments & Research

Please be assured that SMA UK, MDUK and TreatSMA are still actively working on this and continue to ask NICE and NHS England to address the concerns we have at the earliest possible time.

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NICE’s Scoping of Risdiplam Treatment Delayed due to COVID-19

07 April 2020 / Posted in: Treatments & Research

NICE has announced that as it has to prioritise work that is either therapeutically critical or relates to addressing COVID-19 diagnostic or therapeutic interventions, it will not go ahead with this stage of the appraisal process at this time.

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Information Statement about how Access to Nusinersen Treatment will be Affected by COVID-19 (coronavirus)

06 April 2020 / Posted in: Treatments & Research

Advice from clinical experts and discussions with the Patient Groups have informed this statement from NICE, NHS England and NHS Improvement. It concerns those who are already receiving treatment and those who have not yet started treatment.

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Action Taking Place to Address Concerns about NHS and Social Care Covid-19 Policies and Implications for the SMA and Wider Neuromuscular Community

06 April 2020 / Posted in: Information, Support

We are very aware of the concerns there have been, and still are, for all people who have neuromuscular / neurological conditions. Key organisations are voicing and working on these concerns with the Government organisations.

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First Patient in Biogen DEVOTE Trial Treated with Higher Nusinersen Dose

06 April 2020 / Posted in: Treatments & Research

Biogen has announced that they have initiated SMA patient treatment in their DEVOTE study, a Phase 2/3 clinical trial of higher nusinersen doses. Although already approved for use in SMA, there is always room to improve the treatment regime of a successful therapy. With the DEVOTE trial, this is exactly what Biogen are attempting.

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NICE Confirms Appraisal of onasemnogene abeparvovec (Zolgensma) is Defined as Therapeutically Critical

03 April 2020 / Posted in: Treatments & Research

We are pleased to be able to report their decision that the HST evaluation of Zolgensma will progress as planned, assuming the continued availability of staff within the Evidence Review Groups and NICE staff, and that the quoracy of committees are not adversely affected by factors relating to COVID-19.

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SMA Europe Comments on the Recent Positive Recommendation for Zolgensma®

01 April 2020 / Posted in: Treatments & Research

SMA Europe has released this statement re the positive recommendation for the gene therapy treatment, which was released on 27th March by the European Medicines Agency (EMA), and the further information now available about what the licence may make possible.

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Covid-9 Questions from the Community Answered by Neuromuscular Clinicians

30 March 2020 / Posted in: Information, Support

We have some SMA-focused advice on our website from a group of neuromuscular clinical experts who lead the adult and children’s NorthStar and SMA REACH clinical networks and work across a wide range of neuromuscular conditions. All the patient groups have been asking, and continue to ask, more detailed questions which we will update as Q&A pages.

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Zolgensma: Request to NICE to Include Appraisal as a Therapeutically-Critical Topic in COVID-19 Impacted Work Programme

27 March 2020 / Posted in: Treatments & Research

We're aware that due to the challenges of working through the impact of Covid-19 on NICE’s workload, they will need to re-prioritise their HST work programme to focus on therapeutically-critical topics. We have written to them today urging them to ensure this includes the appraisal of this potentially life-changing treatment.

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