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Latest News: Treatments & Research

Map of progress towards global access to nusinersen

05 July 2018 / Posted in: Treatments & Research

We have received an updated map from Biogen which shows the latest status of global access to nusinersen.

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Roche’s Sunfish Clinical Trial – why didn’t it happen here/what about access to future trials?

28 June 2018 / Posted in: Treatments & Research

We asked GOSH if they could throw any light on this and what assurances they could give that the UK won’t miss out in the future. Also, we checked what actions anyone interested in being considered for a clinical trial should take.

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Consultation About Screening For SMA

27 June 2018 / Posted in: Treatments & Research

The National Screening Committee (NSC) has invited us to give comments and feedback on its June 2018 evidence review. We will be collating views on this.

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NICE Committee Meeting

27 June 2018 / Posted in: Treatments & Research

Today, we are at the NICE Committee Meeting in Manchester which is considering whether to recommend access to nusinersen; we will know the outcome in 3 – 4 weeks’ time. Thanks to the other two Patient Representatives who are also attending.

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PTC Therapeutics Present Interim Data from FIREFISH Clinical Trial

21 June 2018 / Posted in: Treatments & Research

PTC Therapeutics recently presented preliminary data from the FIREFISH Phase II clinical trial of their splice-modifying drug, RG7916, at the 2018 Cure SMA conference in Dallas, USA.

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Cytokinetics Release CK-2127107 Phase II Clinical Trial Data

20 June 2018 / Posted in: Treatments & Research

Cytokinetics presented data at the 2018 Cure SMA conference from the Phase II clinical trial with SMA patients of its drug CK-2127107, which has been renamed reldesemtiv.

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Cabinet Secretary for Health and Sport in Scotland Outlines New Pathway For Medicines

19 June 2018 / Posted in: Treatments & Research

A statement came out yesterday that explains how patients with rare diseases in Scotland could get faster access to new treatments following a revised decision-making process. The Scottish Government is introducing a new definition of 'ultra-orphan medicines' that can treat rare conditions.

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Latest Updates on Global Access to Nusinersen

18 June 2018 / Posted in: Treatments & Research

We have asked Biogen to keep us updated with this latest information as we all continue to advocate for broad access in the UK.

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Roche Update: SUNFISH Trial Not Going Ahead In The UK

14 June 2018 / Posted in: Treatments & Research

We have received the following update from Roche about the SUNFISH study, investigating RG7916 in people with SMA Types 2 or 3, which will now not be recruiting in the UK.

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First Cohort Dosing Completed in SRK-015 Phase 1 Clinical Trial

08 June 2018 / Posted in: Treatments & Research

Pharmaceutical company Scholar Rock has announced that dosing of the first cohort has been completed in a Phase I trial of the experimental drug, SRK-015.

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