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Spinal Muscular Atrophy News

Biogen provides community update for SMA Europe

Biogen provides community update for SMA Europe. This update covers recent trial results as well as what’s happening in terms of access in Europe and worldwide, namely: Recent publications and presentations ENDEAR Study Results Early Access Programme Access to reimbursed treatment Registries Please do read it.

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Fast-track scheme for breakthrough drugs from April ’18

The Government has announced a new plan to speed up the time it takes for new life-changing medicines to reach NHS patients.  From April 2018 an expert panel will select around 5 new drugs or devices for fast-tracking, reducing the time taken by the NHS appraisal and approval processes. As part of the plans, the Government…

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Repeat reappraisal plea to NHS England re: EAP for SMA Type 1s

An open letter response has been sent to NHS England, again requesting that the policy on EAP for SMA Type 1 to be reconsidered and restrictions removed to enable all SMA Type 1 babies to have access. The letter drafted by Professor Francesco Muntoni and co-signed by The SMA Trust, along with other patient advocates and…

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SMA Europe holds 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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