Latest News: Treatments & Research

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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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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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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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: 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® (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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