Tel: 01789 267520

Latest News: Treatments & Research

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.

Read full story

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.

Read full story

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.

Read full story

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.

Read full story

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.

Read full story

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.

Read full story

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.

Read full story

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.

Read full story

Roche Community Update: Olesoxime

30 May 2018 / Posted in: Treatments & Research

Due to negative long-term effects, Roche have decided to halt further development of their drug olesoxime. They have provided this community statement.

Read full story

Registration for the Public Gallery for the 27th June NICE Committee meeting

25 May 2018 / Posted in: Treatments & Research

This opens on 30th May and closes 13th June. The meeting will discuss access to nusinersen and be held in Manchester. We understand that there are only 30 places available.

Read full story