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 general news about the work of the DFSG.
The DMD Hub News page from Duchenne UK has all the latest on clinical trials, medical reports and research developments.
Givinostat Update
Givinostat, a potential treatment for Duchenne muscular dystrophy, is being reviewed to see if it should be made available on the NHS in England. A decision hasn’t been made yet, as the committee has asked for more evidence to help them decide.
In clinical trials, givinostat has helped slow down how quickly DMD gets worse. This could make a big difference to children and families living with the condition.
Right now, Givinostat is being reviewed by an expert group from NICE (National Institute of Health and Care Excellence). They help decide which treatments should be available on the NHS in England. After their first meeting on 10 July 2025, they have now asked for more evidence before making a decision. While everyone welcomes the need for strong evidence, this delay adds to the challenges families currently face in accessing treatment.
Usually, after this first meeting that happened on 10 July 2025, NICE either:
· Recommends the treatment for NHS use, or
· Doesn’t recommend it but asks for more discussion.
This time, the committee took a different route. They’ve asked for more evidence before they can decide. This means they haven’t said yes or no yet.
To read the whole article about this, please visit:
https://www.musculardystrophyuk.org/about-us/news
Newborn Screening
The World Duchenne Organization (WDO) is pleased to announce the release of a comprehensive White Paper titled ‘Newborn Screening for Duchenne Muscular Dystrophy: The Time is Now.’
Please access it by clicking on the button below.
White paper on newborn screening for DMD
Three Holiday Venues For The DFSG!
We are delighted to announce that the DFSG will be offering its members three subsidised holidays!
The first is to Trevassack Lake, located on the scenic Lizard Peninsula, in Cornwall. Trevassack Lake is a 26-acre accessible site with holiday accommodation and waterside cafe, dedicated to bringing water-sports and sailing activities to anyone and everyone, regardless of age, ability, disability, or experience and is run by the Children’s Sailing Trust.
The second is to Brickhouse Farm Cottages in Lancashire. Their 12 outstanding, award-winning cottages are set amongst acres of stunning countryside. The cottages vary in size, from 2 to 5 bedrooms, and were purpose-built in 2013 to provide holiday accommodation for families, friends and carers in the most tranquil surroundings of Hambleton yet only a step away from the bright lights of Blackpool!
The third is a return to our very popular holiday venue in Brittany, France. We are delighted to be able to offer a week at Le Fosso, where our DMD families can stay in a converted stone-built Breton farmhouse and cider house. The fully accessible, comfortable, and spacious accommodation is set in six acres of gardens and woodland with various attractions and activities nearby.
All our holidays are open to our members, so if you are currently not a member of the DFSG, please sign up here.
Booking forms will be emailed to our members. Please contact us if you do not receive these booking forms by email.
Phillippa Farrant
It is with great sadness that I am writing to inform you that our Development Officer, Phillippa Farrant, left the Duchenne Family Support Group at the end of September 2022 to go and work for another patient charity within the rare disease community.
I’m enormously grateful to Phillippa for all the work she has put in sustaining DFSG for the last seven years as our Development Officer, and previously as committee member and Chair. She has been tireless in her support of our families and in helping coordinate the committee members and activities. We will miss her enormously.
While we take this opportunity to review our work and priorities, our email address (info@dfsg.org.uk) and office telephone number (0800-121-4518) will be handled by our volunteers and post will continue to be forwarded via our postal address (78 York Street, London, W1H 1DP), so please don’t hesitate to contact us if we can be of help.
Please check your address books and discontinue use of her Eastbourne and Gmail addresses except for personal contact. I’m sure you’ll want to join with us in wishing Phillippa every success and happiness for her future!
Stephen Meech (Chairperson DFSG)
DMD Family Folder: Guide to Adolescence and Adulthood
Congratulations to Duchenne UK on their family folder to help support families through the teenage years and beyond. A big thank you to all who helped on this, including our Development Officer. It is free to download, or a hard copy can be ordered from Duchenne UK. We will have some in the office to send out in the near future.
Download the DMD Family Folder
Jeans for Genes Day
We are pleased to announce that DFSG have received a small grant from Jeans for Genes of £2,500, which will fund our next newsletter and some core costs. These grants are vital to keep our work going.
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.