Latest News: Treatments & Research

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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Planning for Access to Nusinersen – Survey of Adults, What Next

26 March 2020 / Posted in: Information, Treatments & Research, Support

Thanks to all who responded. You can see the ‘top line’ results here. We have forwarded summary information about who has asked for contact to the adult treatment centre network lead clinician. If you gave any personal details but asked for these not to be shared, these have not and will not be passed on.

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SMA Gene Therapy Zolgensma Approved in Japan

24 March 2020 / Posted in: Treatments & Research

Novartis announced on 19th March 2020 that the Japanese Ministry of Health, Labour and Welfare approved Zolgensma for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis. This approval was based on the results from several clinical trials.

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Managed Access Agreement (MAA) for Nusinersen Treatment for patients who have Spinal Muscular Atrophy –plans to manage the impact of COVID-19

20 March 2020 / Posted in: Treatments & Research

NICE and NHS England and Improvement are very aware of the concerns of parents and their children who have SMA, adults who have SMA and clinicians about how COVID-19 is going to affect those who are currently receiving nusinersen treatment as part of the MAA, and the enrolment of anyone further into this programme. They are working hard to get advice around how to manage the safe continuation of current individual treatments.

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Biogen's Statement re: Coronavirus (COVID-19) Outbreak

16 March 2020 / Posted in: Treatments & Research

Biogen have responded to our request for information for the Community regarding the coronavirus (COVID-19) outbreak.

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