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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Changes in Our Activities due to Covid-19

26 March 2020 / Posted in: Information, Support

Our key focus is to keep you up-to-date with information via our website pages and social media. Our Support Services team is here to support you and talk things through. The best way to contact us is via email or phone. We are picking up messages three times a day. Virtual networks continue. Many other activities have had to change.

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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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Roche’s Clinical Development Programme Statement re: Coronavirus (COVID-19) Outbreak

13 March 2020 / Posted in: Treatments & Research

Roche have provided a statement saying they will not exclude patients from SMA clinical studies based on travel restrictions due to the Coronavirus (COVID-19) and will ensure to maintain supply where possible amid the outbreak.

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