Our news page includes a range of articles such as positive news stories from local and national media, opportunities for you to participate in surveys and consultations relevant to people living with Duchenne and reports from our days out (when restrictions are lifted).
The DMD Hub News page from Duchenne UK has all the latest on clinical trials, medical reports and research developments.
The England Rare Disease Action Plan – questionnaire launch (8 November 2021)
About the England Rare Diseases Action Plan
The England Rare Diseases Action Plan will be published to coincide with Rare Diseases Day in February 2022. This Action Plan follows on from the UK Rare Diseases Framework and there will be equivalent plans published from Scotland, Wales and Northern Ireland. It will contain action points from delivery partner organisations such as NHS England, Health Education England, Genomics England and NICE on how we will ensure that the lives of people in England living with rare diseases continue to improve. The Action Plan will be reviewed and updated annually over the five-year course of the Framework.
About the questionnaire
The aim of the questionnaire is to gather feedback from the community on the draft actions that have been developed for inclusion in England’s Action Plan. It is vital to our commitment to put patient voice at the centre of our plan.
The questionnaire is open to all members of the rare diseases community (e.g. people living with a rare disease/ patient or public voice representatives / carers / family members / clinicians / industry professionals / healthcare professionals / researchers) or representatives from organisations involved in rare disease work. Because the questionnaire focuses on England’s action plan, we would ask that you only complete it if you are based in England.
We are particularly seeking input from members of the rare disease community with an interest in rare disease policy. Existing knowledge of the rare disease landscape is desirable (although not essential) for answering this questionnaire.
We estimate it will take around 30-45 minutes to complete. The questionnaire is hosted on the secure DHSC eXchange
Accessing the questionnaire
To access the questionnaire, follow this link: England Rare Diseases Action Plan Questionnaire. The link is public, and we encourage you to share this with others in the rare disease community who may be interested in sharing their views.
WORLD DUCHENNE DAY September 7th 2021
Always an important day in the Duchenne community calendar, this year the theme is focused on Adult Life. So many more of our young people with this condition are living into adulthood and they are an inspiration to us all. Look out for information, videos and photographs from the World Duchenne Organisation.
Making the unseen seen (29 July 2021)
At the beginning of the Covid-19 pandemic of early 2020, a group of concerned advocates came together to take Action for Rare Disease Empowerment (ARDEnt). The ARDEnt group’s aim was to shed light on the unseen impact of the pandemic on people living with rare conditions in order to protect the existing services for people living with rare conditions and lessons can be learned. View the report here: Rare disease and the lessons learned from the COVID-19 pandemic
first UK patient enrolled in DMD gene therapy trial at DMD Hub site (13 May 2021)
We are excited to share the recent news from Duchenne UK, the first UK patient enrolled in a gene therapy trial.
Read all about it here: first UK patient enrolled in a gene therapy trial.