Tel: 01789 267520

Latest News: Treatments & Research

Roche Community Update

16 March 2018 / Posted in: Treatments & Research

Roche has provided the following community statement on RG7916 and Olesoxime investigational programs.

Read full story

Nusinersen Expanded Access Programme for SMA Type 1 Update

12 March 2018 / Posted in: Treatments & Research

NHS England has now agreed to fund all children with SMA Type 1 who have been diagnosed by age 7 months, irrespective of the number of SMN2 copies they have. This is good news and our thanks go to Professor Francesco Muntoni who has tirelessly led the advocacy for this.

Read full story

What is the Likely Timeline for any Decisions about Access to Nuisnersen in England?

28 February 2018 / Posted in: Treatments & Research

We have had a number of queries about what is happening about any Managed Access Agreement and decisions from NICE. You can read our understanding of what is most likely, here.

Read full story

SMA Support UK at the International Scientific Congress on SMA 2018

27 February 2018 / Posted in: Treatments & Research

Organised by SMA Europe with Brunhilde Wirth as the Scientific Chair, the first International Scientific Congress on SMA was held at Jagiellonian University in Kraków, Poland from 25th-27th January 2018. More than 100 laboratory and clinical SMA studies were presented by researchers from across the world to over 450 attendees.

Read full story

Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research

21 February 2018 / Posted in: Treatments & Research

Biogen has provided a community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA.

Read full story

Are you Affected by SMA Type 1 and Willing to Share your Experience with TreatSMA?

16 February 2018 / Posted in: Treatments & Research

As part of our ‘working together for access to nusinersen’, TreatSMA is gathering people’s experiences to present to NICE. If you are willing to take part, please click here to find out more.

Read full story

Are you Willing to Talk to the Media About your Experience of SMA and Access to Nusinersen?

16 February 2018 / Posted in: Treatments & Research

As part of our ‘working together for access to nusinersen’, MDUK is managing our media contact & would like to hear from you. You'd be fully supported by a member of the MDUK media team through the interview process, & you'd get to choose which media you're happy to speak to.

Read full story

Rare Disease Day – 28th February 2018

14 February 2018 / Posted in: Treatments & Research

Taking place each year on the last day in February, the main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives.

Read full story

Map the Gap Campaign

14 February 2018 / Posted in: Treatments & Research

Part of the larger initiative, The GenomeAsia100K project, Map the Gap aims to sequence 100 South Asian Genomes, enabling the development of carrier tests, diagnosis, and treatment for rare diseases, cancers, and diabetes.

Read full story

Opportunity to let NICE hear your views about the impact of 5q SMA and who should be able to access nusinersen

12 February 2018 / Posted in: Treatments & Research

SMA Support UK has set up a number of surveys so that we can collect different people’s views and share them with MDUK, SMA Trust, TreatSMA, and NICE. Deadline to complete survey(s) is 18th Feb.

Read full story