NICE Announces Guidance on Nusinersen will be Published on 26th June

15 May 2019 / Posted in: Treatments & Research

NICE have now updated their website with this advice. This means that until 26th June, Biogen will fund currently untreated infants with SMA Type 1 via a “bridging solution”. All patients already on the EAP will continue to have access to treatment. Those with SMA Types 2 and 3 will then start to get access subject to the terms of the MAA and as soon as services are able to cope with the patient load.

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NHS England Announces Access to Nusinersen for all with SMA Types 1, 2, 3a and 3b

15 May 2019 / Posted in: Treatments & Research

The treatment will be made available by Biogen immediately to the youngest and most severely-affected infants (SMA Type 1). The NHS will then begin to provide nusinersen to those with SMA Types 2 and 3 shortly after NICE’s full guidance is published, and once the services to deliver them are established. It will also be available to pre-symptomatic siblings who, based on their genetics, are very likely to develop SMA.

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Mental Health Awareness Week 2019

14 May 2019 / Posted in: Information, Support

This week is Mental Health Awareness Week. Read Rare Disease UK's report from 2018 about mental health and the impact of living with a rare disease, and find out more about how we may be able to help with emotional support.

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Potential Biomarker for SMA Identified

10 May 2019 / Posted in: Treatments & Research

Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA.

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Pre-symptomatic Infants Treated with Spinraza™ Achieve Motor Milestones More Consistent With Normal Development

10 May 2019 / Posted in: Treatments & Research

Data from the NURTURE study showed that pre-symptomatic treatment of infants very likely to develop SMA Type 1 or 2 due to their inherited genes resulted in motor milestone achievements more consistent with normal motor development.

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AveXis Presents Positive Interim Results on Zolgensma Across a Broad Spectrum of Patients with SMA

07 May 2019 / Posted in: Treatments & Research

AveXis presented positive results from clinical trials on Zolgensma, across a broad spectrum of patients with SMA at the 2019 American Academy of Neurology conference in Philadelphia.

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Data Published in Neurology Suggest Long-Term Benefits of Spinraza™ Treatment in Later-Onset SMA

07 May 2019 / Posted in: Treatments & Research

Biogen has announced that data from a study on the safety and tolerability of Spinraza™ in individuals with later-onset SMA had been published in the peer-reviewed journal, Neurology.

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Education Secretary Seeking Views On Funding For Children With Special Needs

03 May 2019 / Posted in: Information, Support

Damian Hinds is at a conference of the National Association of Head Teachers today where he is expected to make an announcement calling for a fresh look at educational funding for children with special needs in England.

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Response From Biogen to SMA UK & MDUKs' Letter

03 May 2019 / Posted in: Treatments & Research

Following SMA UK and MDUK sending letters urging Biogen, NHS England and NICE to work strenuously this week so that the NICE committee can endorse an agreement for access to nusinersen for all with SMA Types 1, 2 & 3, we have received a response from Biogen.

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SMA UK and MDUK write to NICE, NHS England and Biogen

02 May 2019 / Posted in: Treatments & Research

Read the flyer and letters that were sent urging these three organisations to work strenuously this week so that on May 8th, the NICE committee can endorse an agreement for access to nusinersen for all with SMA Types 1, 2 and 3.

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