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Give as you Live™? Every purchase you make online can raise funds for DFSG, at no extra cost.Using 'Give as you Live' is a really easy way to help us raise funds for DFSG. Use the link to go shopping and help support DFSG. Some retailers give up to 3% commission and in December 2015 DFSG made £150 from 'Give as you Live'.

dfsg-logo.jpgThe Duchenne Family Support Group - DFSG

The Duchenne Family Support Group (DFSG) exists to provide support for families with children diagnosed as having Duchenne Muscular Dystrophy (DMD).

The DFSG is a national charity run by families for families affected by DMD. It provides a positive national support network of parents, their families and professionals.

The DFSG helps bring families together for mutual support, sharing of information and experience, and social activities and has been doing so since 1987.

Fundraise for Duchenne Family Support Group when you shop online

Fundraise for Duchenne Family Support Group when you shop online

The London Marathon

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The 2018 Virgin Money London Marathon took place on 22nd April 2018. A healthy sum was raised through the efforts of our runners for which we say a heartfelt 'thank you' although our numbers were depleted due to injury. We are looking to 2019 when the marathon is scheduled for Sunday 28th April 2019. There are seven places for runners available, please contact DFSG if you are interested in participating

 

An announcement on the Early Access to Medicines Scheme for RaxoneThank You - An announcement on the Early Access to Medicines Scheme for Raxone

We are delighted to announce that the MHRA have decided to maintain a positive opinion on the Early Access to Medicines Scheme for Raxone to treat people living with Duchenne muscular dystrophy. This means that Raxone remains on course to have its EAMS award renewed, so that patients over 10 years of age, showing a decline in respiratory function and who are currently not taking glucocorticoids, can continue to access Raxone until June 2019

This was a joint effort from the entire Duchenne community and Action Duchenne, DMD Pathfinders, Duchenne Family Support Group, Duchenne UK and Muscular Dystrophy UK cannot thank enough, all of you who provided vital feedback to this process.

Each and every organisation appreciates how much effort and courage goes into providing this testimony, whether it is delivered in writing or in person, and we have no doubt this had a positive impact on the committee’s decision.

The UK Commission on Human Medicines (CHM) determined from the evidence and testimony received, that the risk/benefit profile was acceptable and there is a clear high unmet medical need for patients with Duchenne. Receiving feedback from such a wide spectrum of the community was integral to this.


"DFSG is delighted with the positive news that the EAMS scheme for Raxone is being renewed based on scientific opinion of the risk/benefit ratio. The MHRA involved Action Duchenne and all the UK patient organisations in this process. As a collective powerful UK community, we supported young people, adults living with Duchenne and caregivers both in collecting vital lived experience testimonies on respiratory health and in person,” said DFSG Development Officer Phillippa Farrant “Raxone is available for those who are eligible and after appropriate medical consultation
The Early Access to Medicines Scheme (EAMS) for Raxone

  • The aim of the EAMS is to provide earlier availability of promising medicines to patients in the UK where there is high unmet clinical need.
  • It is to slow the decline of respiratory function in patients with Duchenne from the age of 10 years who are currently not taking glucocorticoids.
  • At the same time more data is able to be captured, to evaluate the effectiveness of the medicine.
  • Treatment will only be provided in specialist centres that are experienced in managing Duchenne and there is the requirement for careful monitoring to ensure the safety of patients during the EAMS.
  • People with Duchenne may discuss together with their doctor whether they are suitable for this treatment.

This Frequently Asked Questions document provides more detail, but if you have any questions please do not hesitate to contact us.

 

Recent News, Articles and Conferences

Adult Duchenne guidelines consultation period open

Adult Duchenne guidelines consultation period open

Friday 27th October 2017 Muscular Dystrophy UK brought together adults living with Duchenne muscular dystrophy, health professionals, partner charities and pharmaceutical representatives to discuss new best-practice care recommendations being created for health professionals supporting an adult with Duchenne in the UK.

Prudential RideLondon 2017

Prudential RideLondon 2017

Developed by the Mayor of London and his agencies in 2013, Prudential RideLondon is a world-class festival of cycling. The fifth edition took place over the weekend of 28-30 July 2017. The event features the Prudential RideLondon-Surrey 100 – a 100-mile challenge which three cyclists rode in support of DFSG

New information to help families from Muscular Dystrophy UK

New information to help families from Muscular Dystrophy UK

Muscular Dystrophy UK has launched the second edition Adaptations manual: for children and adults living with muscle-wasting conditions. Families regularly approach Muscular Dystrophy UK with questions about the best adaptations to get for themselves and for family members. The second edition of the adaptations manual aims to answer those questions.

TEN POSTS IN PLACE TO SUPPORT UK CLINICAL TRIAL CAPACITY

TEN POSTS IN PLACE TO SUPPORT UK CLINICAL TRIAL CAPACITY

In December 2015, eight patient organisations, who are working to help support clinical trial development for Duchenne muscular dystrophy, announced a total of £1.2million of investment, to fund a total of 16 clinical posts. Today we are announcing that TEN of those SIXTEEN posts have now been recruited. The posts are at Great Ormond Street Hospital NHS Foundation Trust in London (GOSH), The John Walton Muscular Dystrophy Research Centre at Newcastle University, University Hospitals Bristol NHS Foundation Trust and Alder Hey Children’s Hospital in Liverpool.

The Newcastle  Plan

The Newcastle Plan

Charities announce £1.2million investment in key clinical posts at leading Neuromuscular Centres for Duchenne Muscular Dystrophy. Eight patient organisations, who are working to eradicate Duchenne Muscular Dystrophy, are funding a total of 16 clinical posts at Great Ormond Street Hospital in London, The John Walton Muscular Dystrophy Research Centre at Newcastle University and Alder Hey Children’s Hospital in Liverpool, to help support clinical trial development for Duchenne Muscular Dystrophy. The DFSG are pleased to be involved with this project and although not supporting financially as this is not in our remit, we are working closely with all of the charities in a n advisory capacity .

NICE has recommended funding on the NHS in England for Translarna.

NICE has recommended funding on the NHS in England for Translarna.

NICE has recommended funding on the NHS in England for Translarna. This historic decision would make available on the NHS the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy. Translarna would treat boys whose Duchenne is caused by a nonsense mutation, who are aged five years and over and who are still able to walk. In order to give time for NHS England and the drug company, PTC Therapeutics, to agree an final acceptable price, NICE has set July as the date where its recommendation will become final. We’ll then be pushing NHS England to implement this decision in order to get the drug to boys in clinic as quickly as possible.

SKIP-NMD – a new Duchenne clinical trial starting

SKIP-NMD is funded by the European Seventh Framework Programme FP7 and involves research institutions and muscle centres from France, Italy and the UK. The aim of the consortium is to carry out a new clinical trial to test a molecular patch for exon 53. This is to restore dystrophin production in a subset of boys with Duchenne muscular dystrophy, in particular those with a deletion (a mutation where part of the dystrophin gene is missing) spanning exons 52, 45-52, 47-52, 48-52, 49-52 and 50-52. Read More...

SKIP-NMD reports progress on skipping exon 53

Encouraging results on the successful completion of the first part of a clinical trial to skip exon 53 in boys with Duchenne muscular dystrophy have been reported at the SKIP-NMD Consortium meeting that took place in Sardinia from the 24-25 June 2015. Full report published here...

Download the pdf

SKIP-NMD news article | SKIP-NMD a new Duchenne clinical trial starting | The SKIP-NMD website


Holidays and Special Events

Hall Farm Cottages Norfolk August 2018

Hall Farm Cottages Norfolk August 2018

HALL FARM COTTAGES, HORNING, NORWICH, NR12 8NJ. SATURDAY, 18TH AUGUST to SATURDAY 25TH AUGUST 2018. We are pleased to be able to offer our families another holiday in 2018 in these wheelchair friendly self-catering holiday cottages in Norfolk. You can find lots of information on their website www.hallfarm.com. Hall Farm Cottages are fully accessible. They are dog friendly (extra charge) and situated a short drive from Wroxham on the Norfolk Broads.

DFSG Day Trips

DFSG organises a number of accessible day trips from time to time. We have three in August this year to Beaulieu, Duxford and Chester Zoo.

2018 visit To Le Fosso France

2018 visit To Le Fosso France

The 'Le Fosso Trip' 2018 has taken place and was a very exciting and successful holiday for everyone. Full report and photos to follow along with the dates for 2019 when they become available. Enquiries to phillippa.farant@dfsg.co.uk

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Review of 2015 trip to Le Fosso in Brittany by Penny Bailey

This is the second year that we attended the trip to Le Fosso with DFSG and what a week we had! Fantastic weather, meeting up with familiar faces and enjoying the hospitality of Mike and Hilary (oh, and yes, catching up with the animals!).

Read more...

Willow Foundation

Willow is the only national charity working with seriously ill young adults aged 16 to 40 to fulfill uplifting and unforgettable Special Days that enable them and their families to reconnect and refocus on each other while enjoying an activity of their choosing. A day that will create memories they will all treasure forever. - See more at: www.willowfoundation.org.uk


Living with DMD

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Holiday News

Wheelchair Accessible Motorhome

Fully wheelchair accessible motorhome

Road Runner Motorhomes is a family run, Community Interest Company (CIC), offering disabled people the opportunity to hire a specially equipped, fully wheelchair accessible motorhome at affordable rates. Their aim is to make going on holiday both accessible and achievable for those with a disabled family member. The 4 berth motorhome features: entry via an electrically operated cassette wheelchair lift, a four point wheelchair tie-down system, a profiling bed, an overhead hoist with tracking from the profiling bed to a wet room and a 240 volt inverter to charge a wheelchair and/or run a Bi PAP.
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