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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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 Not Defined as Therapeutically Critical

03 April 2020 / Posted in: Treatments & Research

NICE has decided that the HST evaluation of onasemnogene abeparvovec is not defined as therapeutically critical. The HST evaluation will progress as planned with company and Evidence Review Group input into the next phase of the timeline. NICE will provide further updates on the progress of the evaluation when known.

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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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Zolgensma data shows rapid, significant and clinically meaningful therapeutic benefit in patients who have SMA

27 March 2020 / Posted in: Treatments & Research

Avexis provide an update on several of their clinical studies showing that a single treatment with Zolgensma (onasemnogene abeparvovec) can result in rapid, significant and clinically meaningful therapeutic benefit in people with SMN-linked SMA. This effect was sustained in patients who received their treatment up to five years ago, some of whom are now more than five years of age.

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Zolgensma® (onasemnogene abeparvovec) Recommended for European Commission (EC) Conditional Approval

27 March 2020 / Posted in: Treatments & Research

This is for patients who have SMA and a clinical diagnosis of Type 1 or SMA patients with up to three copies of the SMN2 gene.

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Study Shows Nusinersen is Safe and Effective for SMA Treatment in Adults

27 March 2020 / Posted in: Treatments & Research

A study, published in the Lancet this month, has shown nusinersen to be safe and effective for the treatment of SMA in adults. Dr. Hagenacker and team carried out an observational study of 173 adults with SMN-linked SMA at ten academic clinical sites in Germany.

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Our Supportive Board of Trustees

26 March 2020 / Posted in: Information, Support

We've had great support during these difficult days from our Trustees. They know our main focus is to provide information and support to the Community, but with fundraising events cancelled and everyone needing to look after their own needs first, they're also doing their best to help us plan round a massive drop in income and the impact this will have on our staff and capacity to keep going.

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