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'.
The 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.
The 2020 London Marathon
The 2020 Virgin Money London Marathon takes place on 26th April 2020. We have a few spaces available for those who would like to take part in the 2020 London Marathon and raise money for DFSG at the same time. In previous marathons healthy sums have been raised through the efforts of our runners for which we say a heartfelt 'thank you' although our numbers were depleted due to injury. Please contact DFSG if you are interested in participating.
Thank 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.