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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Survey Results Update from OpenVie on behalf of Roche

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

Thank you to everyone who completed this survey. Read more about how they will use the data, including writing a report to share with UK Health Technology Agencies.

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Shift Pharmaceuticals Receives US FDA Orphan Drug Designation for their Lead Compound for SMA

09 March 2020 / Posted in: Treatments & Research

Orphan Drug Designation gives incentives to companies to develop drugs intended to treat rare conditions, such as SMA, and should help to expedite the development of E1v1.11 as a potential therapeutic option for SMA.

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