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Spinal Muscular Atrophy News

NHS England Announces Interim Policy on EAP for SMA Type 1

NHS England has considered the evidence available within the European Appraisal and have today published an interim policy providing all hospital costs for administering nusinersen to patients with SMA Type 1. This is to support the access scheme offered by Biogen and will continue until either the company cease the access scheme to new patients or when the appraisal…

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Biogen: EAP for SMA Type 1 is planned to stay open until year end

In response to a request from SMA Trust and SMA Support UK, Biogen has announced it’s current intention to keep the EAP for SMA Type 1 open ‘for the remainder of the year in the UK’ and will continue to ‘engage with NHS England on the EAP in England and with NICE on the reimbursement pathway…

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The SMA Trust attends the Cure SMA’s Annual Conference in Orlando

Cure SMA’s annual conference took place at the Disneyland Contemporary Hotel (Disney World), in Orlando, Florida, between 29th June and 1st July. This conference really consists of two main events: the families’ meeting and the researchers’ conference. Joanna, CEO and Vanessa, Research Coordinator, attended the researchers’ conference, as well as a series of satellite meetings…

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SMA Europe awards new research grants through its 9th call for research proposals

SMA Europe awards new research grants, providing vital funds to SMA research. Based on the recommendations received by its Scientific Advisory Board, SMA Europe awarded a total of €357,800 to 3 cutting-edge SMA research projects through its 9th Call for Research Proposals. The Scientific Advisory Board reviewed and scored 21 very high level SMA research…

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Revised Hammersmith Scale for SMA: paper published!

What is the Hammersmith Scale? The Hammersmith scale is a well recognised functional scale used clinically as well as in clinical trials across the world to measure physical abilities of children and adults with SMA type 2 and 3. Background Recent translational research developments in SMA, outcome measure design and demands from regulatory authorities require…

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UK SMA Research Consortium holds national research meeting in Oxford

The UK SMA Research Consortium held a research meeting last month with the aim of bringing other UK groups into the project. Industry representatives were also present. The event took place at St. Hugh’s College in Oxford on 13th June and involved Consortium investigators and guest speakers from national research groups presenting their work to…

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Scholar Rock to start clinical trials in SMA

Scholar Rock announced last week that SRK-015, the company’s lead clinical candidate, will be developed to improve muscle strength and function in patients with SMA. What is Scholar Rock? Scholar Rock is a biotechnology company which specialises in the discovery and development of innovative new medicines to treat a range of serious diseases with high…

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Biogen statement following disclosure of EU list price for nusinersen

In response to a request from SMA Europe, Biogen representatives and the board members of SMA Europe participated in a joint conference call on Friday 9th June, 2017 to discuss the EU list price for nusinersen. Following a subsequent request to share a summary of the discussion, Biogen provided the following update. For more information…

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Update from NHS England meeting about the SMA Type 1 EAP

Lead clinicians, centre service managers and SMA Support UK attended a meeting on 6th June on the expanded access programme (EAP) for nusinersen. Due to the election ‘purdah’ and the NHS England’s (NHSE) communications approval process, it has not been possible to report on the outcome of this meeting until the release of the meeting…

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