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Spinal Muscular Atrophy News

SMA Europe held its bi-annual meeting in Berlin

SMA Europe held its bi-annual meeting in Berlin over 13th and 14th October 2017, kindly hosted by Initiative SMA, an initiative of the German neuromuscular organisation, Deutsche Gesellschaft für Muskelkranke (DGM). As a member country, The SMA Trust was present to represent the UK, along with SMA Support UK. A number of topics were discussed, from the…

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Roche provides community statement regarding SUNFISH

Roche provides community statement regarding SUNFISH, a clinical trial of RG7916 for SMA Types 2/3. An interim analysis was recently presented at the International Conference of the World Muscle Society in France. In this analysis, all participants (51 people) had received RG7916 for 28 days or longer. SMN protein increased by up to two and…

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AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1

The U.S. Food and Drug Administration (FDA) has notified AveXis that, based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with SMA Type 1 using the intravenous (IV) formulation produced by the company’s Good Manufacturing Practice (GMP) commercial manufacturing process. The company plans…

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Unsatisfactory response from NHS England re: criteria for EAP

We have now had a reply to the joint letter which was sent from clinicians and patient advocacy groups to NHS England in August, requesting broadening of the criteria for Type I babies to be included in the EAP.  Unfortunately they are not able to change the current ruling so any new decision would need…

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Request to NHS England re: interim policy on EAP for SMA Type 1

An open letter has been sent to NHS England asking for the policy on EAP for SMA Type 1 to be reconsidered and restrictions removed with respect to age and eligibility criteria. The letter co-signed by The SMA Trust, along with other patient advocates and clinicians can be read in full below: Nusinersen letter August 2017

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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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