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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SMA UK Family Meet-Up: South East Rearranged Due To Coronavirus

06 March 2020 / Posted in: Information, Support

Due to the escalation of COVID-19 coronavirus, we have taken the difficult decision to rearrange this event to Sunday 25th October. We hope to go ahead with our other planned events which are scheduled to take place later in the year.

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Update on AveXis Global Managed Access Programme (GMAP) for Zolgensma

05 March 2020 / Posted in: Treatments & Research

AveXis started to roll out this programme for children age under 2 years who have SMA in January 2020. SMA Europe was one of many groups to express concerns about the programme’s design. Read more about the UK’s current position on this following discussions between the SMA REACH network of clinicians and patient group representatives, and AveXis UK, the Medicines and Healthcare Products Regulatory Authority (MHRA) and NHS England.

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Roche Completes Recruitment for JEWELFISH Clinical Trial

28 February 2020 / Posted in: Treatments & Research

Roche has shared a community update to inform us about recruitment completion for their JEWELFISH clinical trial. This milestone marks the enrolment of 174 participants from across the globe.

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Day-to-Day Activities Survey for People who have SMA Type 2 or 3 and their Unpaid Parents / Carers

28 February 2020 / Posted in: Information, Support

This study is looking for a limited number of adults and parents / caregivers of teenagers or children to complete a 60-minute online survey. If you are eligible and fully complete the study, you will receive £100 compensation for your time.

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