News
The latest updates on our research projects and fundraising achievements.FDA approves first gene therapy for Duchenne
The FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world …
DRF highlights psychosocial research at parent information day
The DRF was pleased to participate in Duchenne UK’s latest parent information day, held in March in central London.
See the smart suit that is changing children’s lives
On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart suit’ aims to help children who are losing upper body function in still continuing to use their arms.
DRF co-founder raises £15,000 in first marathon
We are so proud of our co-founder, Doron Rosenfeld, who has completed his first ever marathon in 4 hours 43 minutes.
DRF-funded KineDMD study published in Nature Medicine and featured on BBC
We are delighted that the ground-breaking KineDMD activity monitoring study we funded has been published in the prestigious scientific journal Nature Medicine, and covered by the BBC …
Solid completes acquisition of AavantiBio
We are pleased to share that Solid Biosciences has completed its acquisition of gene therapy company AavantiBio in a pivotal moment for the company …
Parents urged to read new cardiac recommendations for Duchenne
DMD Care UK’s new cardiac care recommendations have been published in the BMJ Open Heart journal. All Duchenne parents and caregivers are being urged to read the new guidance and share with their networks, as protecting the heart early is crucial to preventing unnecessary deaths. Leading cardiologists, Duchenne specialists and patients and caregivers came together …
Solid to acquire AavantiBio and focus on next-generation gene therapy candidate
Solid Biosciences has announced its entry into a definitive merger agreement to acquire AavantiBio, to create a genetic medicines company focused on neuromuscular and cardiac rare diseases, maintaining a strong focus on Duchenne …
DRF-funded physio role to become permanent at GOSH
We are delighted to share that Great Ormond Street Hospital Trust has agreed to make permanent a physiotherapist role that the Duchenne Research Fund has funded for the past few years …
Psychological and psychiatric support put in place for Duchenne patients and families
Dedicated specialist support for Duchenne muscular dystrophy (DMD) patients and families’ mental health and wellbeing will soon be more readily available, as part of the DMD Care UK project.
DMD Hub launches Central Recruitment Pilot Project
The DMD Hub is launching the Central Recritment Pilot Project, aiming to create a database of families interested in take part in clinical trials …
Solid data shows improved motor and lung function
We are pleased to share Solid Biosciences' latest update on the IGNITE DMD gene therapy clinical trial. Data after two years suggests that patients' motor function and lung function are improved compared to the declines that would be expected without treatment. The...
Emergency app launched for Duchenne
An ‘In Case of Emergency’ app has been launched for Duchenne patients and their families, as part of the DMD Care UK project …
DRF-funded project aims to screen for neurobehavioural issues
The Duchenne Research Fund is pleased to share an update on the PPMD project we have funded evaluating neurobehavioural and psychosocial issues in Duchenne. Analysis of the data is ongoing, but key findings include …
Five-year grant to Dr Michela Guglieri: outcome report
We are delighted to report the outcomes of our grant for a Clinical Trials Lectureship. The grant, which was supported by a consortium of seven UK charities …
DMD Care UK: Psychosocial Programme now recruiting
We are pleased to announce that the University of Newcastle is now recruiting for a clinical psychologist to work with Duchenne patients …
Santhera and ReveraGen share latest vamorolone study results
We are pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma of new topline results after completion of the VISION-DMD study at week 48 …
DRF awards £640,000 for vital psychosocial project
The Duchenne Research Fund is delighted to announce that we are awarding a £640,000 grant to fund vital psychosocial care and research as part of Duchenne UK’s DMD Care UK programme.
#TeamDRF raise £28,000 in London Marathon
Congratulations to #TeamDRF's Adam, Benji, Charles and Yossi who completed the London Marathon on Sunday 3rd October 2021, raising more than £28,000 for the Duchenne Research Fund. The annual event is the largest marathon in the UK, attracting around 40,000...
DRF-funded drug shows success in clinical trial
We are pleased to share that Vamorolone, an alternative medicine to corticosteroids, has shown positive results in a Phase 2 Study …
Watch the highlights of An Evening with the DRF
Watch the highlights of Gavriel Rosenfeld in conversation with Dr Ronni Cohn at our virtual Evening with the Duchenne Research Fund. More than 500 people watched our virtual event to hear Gavriel talk about living with Duchenne, mental health, juggling school and hospital visits, and more …
Gavriel Rosenfeld talks to Dr Ronni Cohn about living with Duchenne
On Wednesday 17th March the Duchenne Research Fund hosted a virtual evening giving supporters an update on ongoing research funded by the charity. We were so thrilled that Gavriel Rosenfeld, the son of the DRF’s founders Kerry & Doron Rosenfeld, joined the virtual event …
DRF funds PPMD neurobehavioural research
We have awarded an £80,000 grant to Parent Project Muscular Dystrophy (PPMD) to support its research into neurobehavioral issues in Duchenne – focusing on key areas such as learning, behaviour, depression and anxiety …
DRF funds Duchenne clinic at Great Ormond Street
We are pleased to share that we have awarded a grant of £35,000 to Great Ormond Street Hospital to establish a dedicated clinic for children who are non-ambulant (no longer able to walk). The grant supports the post of Senior Physiotherapist Nicola Burnett and has enabled more than 80 patients to be seen …
DRF grants £40,000 to DMD Care UK project
We have awarded £40,000 to the newly launched DMD Care UK, partnering with the project’s founders Duchenne UK and Joining Jack. Together our three charities will invest £130,000 in the nationwide initiative that aims to ensure the best care for everyone living with Duchenne in the UK …
Run the London Marathon with DRF
We are pleased to announce that the Duchenne Research Fund has places available for the 2021 Virgin Money London Marathon, which is due to take place on Sunday 3rd October …
ReveraGen receives $3.3m NIH grant for Vamorolone
ReveraGen BioPharma has received a $3.3 million grant from the NIH – the US National Institutes of Health – for their work on Vamorolone …
Solid announces collaboration with Ultragenyx
Ultragenyx Pharmaceutical and Solid Biosciences have announced a strategic collaborations and license agreement to focus on the development and commercialisation of new gene therapies for Duchenne …
Two-year Vamorolone study completed
Santhera Pharmaceuticals has announced that partner ReveraGen Biopharma has completed a long-term, open-label extension study of 24 months duration with Vamorolone in patients with Duchenne…
Update on Ignite DMD from Solid Biosciences
Solid Biosciences has issued an update on IGNITE DMD, the Phase I/II clinical study with its microdystrophin gene therapy, SGT-001.
DRF-funded research featured on ITV’s Little Heroes
We are so proud that the family of our founders, Kerry and Doron Rosenfeld, starred in Paul O’Grady’s Little Heroes on ITV …
DRF-funded Imperial College AI study extended
The Duchenne Research Fund is pleased to share that the ground-breaking KineDMD activity monitoring study at Imperial College London/GOSH has been extended into 2021…
Cyclists take on Prudential ride to raise funds for DRF
Congratulations to the #TeamDRF cyclists who once again took on the Prudential RideLondon-Surrey, and raised more than £5,000…
Martyn and Jessica trek Snowdonia to raise funds for DRF
Congratulations to Jessica and Martyn Jacobs who trekked Snowdownia to support the Duchenne Research Fund …
Sam and Avi run marathon for DRF
Thank you to Sam Baum, Avi Mocton and Alex Klinger who raised funds for the Duchenne Research Fund …
DRF funds University of Florida research to tackle gene therapy immunity challenges
The Duchenne Research Fund has awarded a grant to Dr Barry Byrne and Dr Manuela Corti’s team at the University of Florida, whose vital work focuses on overcoming immunological challenges to ensure all boys and young men can benefit from gene therapy treatments for...
DRF Head of Scientific Advisory Board becomes hospital President and CEO
We are delighted to share the news that Dr Ronald Cohn, the head of the DRF’s Scientific Advisory Board, has been made President and CEO of the Hospital for Sick Children …
Solid Biosciences announces preliminary gene therapy clinical trial results and intention to dose escalate
Solid Biosciences has announced preliminary data from its gene therapy clinical trial, IGNITE DMD …
DRF funding helps double number of children on neuromuscular clinical trials at GOSH
We are pleased to share a report from Great Ormond Street Hospital (GOSH) about the positive impact of the clinical staff posts that we helped to fund at the hospital, together with Duchenne UK, Harrison’s Fund and the Great Ormond Street Charity …
DRF co-founders honoured by Prime Minster’s Office
The Duchenne Research Fund’s co-founders Kerry and Doron Rosenfeld are honoured to have received the Prime Minister’s Points of Light award …
Lauren skydives in memory of brother Charlie
Thank you to Lauren Halsey who raised funds for the DRF by skydiving …
DRF funds CRISPR research to correct Duchenne mutations
The Duchenne Research Fund has awarded a £200,000 grant to the team at the Hospital for Sick Children in Toronto, Canada (‘Sick Kids’), who are working on using CRISPR/Cas9 technology…
#TeamDRF raises £30,000 in Prudential cycle ride
Congratulations to the #TeamDRF cyclists who took on the Prudential RideLondon-Surrey on Sunday 29 July in treacherous conditions and raised more than £30,000…
Vamorolone shows improvements in strength and endurance in boys with Duchenne
We are pleased to share an update from ReveraGen about its Vamorolone clinical trial. Vamorolone is an alternative treatment to existing steroids that aims to maximise the benefits while reducing the side effects. In 2014, The Duchenne Research Fund helped fund the...
Gene therapy for Duchenne: latest news
Five years ago we identified gene therapy as a viable treatment for Duchenne. Today we are reflecting on the exciting and inspiring events of the past week in the gene therapy arena which are now bringing hope and cautious optimism to the Duchenne community. We have...
Solid Biosciences clear to resume IGNITE DMD
Message from Solid Biosciences: "We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD, our clinical trial investigating SGT-001 as a potential treatment for Duchenne muscular dystrophy (DMD). SGT-001 is a microdystrophin gene...
DRF-funded Imperial College AI study aims to accelerate Duchenne research and trials
The Duchenne Research Fund recently shared the news that we are supporting the KineDMD study - Dr Aldo Faisal's ground-breaking artificial intelligence study at Imperial College London, in collaboration with Great Ormond Street Hospital, which aims to accelerate...
DRF funds Newcastle refurbishment
On Wednesday 27 September 2017, the newly refurbished paediatric area in the Clinical Research Facility at Newcastle Royal Victoria Infirmary was officially opened by Professor Mark Walker and Dr Michela Guglieri.
Duchenne Hub day tackles clinical trial access
The Duchenne Research Fund was delighted to participate in the Duchenne Hub information day held on 18 September 2017 at Alder Hey Children’s Hospital in Liverpool.
Solid Biosciences news
Solid Biosciences recently reported that it has raised up to $50 million which will enable the biotech to continue to advance its programs for Duchenne muscular dystrophy, including development of a microdystrophin gene. We are forever grateful to our incredible...
DRF raises £140,000 in Yorkshire Three Peaks challenge
On Sunday 9th July 2017, 43 intrepid hikers from London, Manchester and New York donned their walking boots and waterproofs and braved the challenging Yorkshire Three Peaks: a 40km hike with 1600 metres of ascent in a target time of 12 hours, raising money to support vital Duchenne Research Fund projects …
Vamorolone receives Fast Track designation from FDA
We’re pleased to share news from ReveraGen, the company developing the novel steroid alternative Vamorolone (previously called VBP15), that their drug has received Fast Track designation from the FDA, which will speed up the review of Vamorolone.
DRF supports projects to tackle fibrosis and enhance drug development
The Duchenne Research Fund is delighted to share the details of the exciting new projects we are funding thanks to donors’ generosity and support, including tackling fibrosis and inflammation, and developing non-invasive biomarkers.
ReveraGen BioPharma licenses option to develop Vamorolone with Actelion
We’re sharing news that ReveraGen BioPharma has licensed an option to develop Vamorolone with Actelion Ltd.
Vamorolone is the first in-human steroid like compound that has shown in animal models to have many of the benefits of corticosteroids, without the many side effects …
DRF marks World Duchenne Awareness Day
On Wednesday 7 September 2016, the Duchenne Research Fund team, together with the charity’s co-founder Kerry Rosenfeld and her son Gavriel, marked World Duchenne Awareness Day.
The theme for 2016 is early diagnosis: focusing on how identifying and communicating early signs of Duchenne muscular dystrophy …
Duchenne patients recruited for steroid replacement study
We are delighted to announce that ReveraGen BioPharma, the company developing a steroid replacement for Duchenne muscular dystrophy, has begun recruiting patients for its Phase 2a open label study. The trial will be recruiting patients who haven’t yet started steroids, between the ages of 4 and 7 …
Ten posts in place to support UK clinical trial capacity
Today we are announcing that TEN posts have been filled out of sixteen clinical posts to support clinical trial development for Duchenne. The posts are part of a £1.2m investment by eight patient organisations under the Newcastle Plan.
DRF raises £1.2 million to help beat Duchenne
The Duchenne Research Fund raised £1.2 million at its ‘The Time Is Now’ Gala Dinner on 5 May at the Lancaster London Hotel to help find a cure for Duchenne muscular dystrophy …
DRF-funded drug development programme receives further £4.6 million
The Duchenne Research Fund, the Duchenne Children’s Trust and Joining Jack and are delighted with the news from the John Walton Muscular Dystrophy Research Centre that they have received £4.26 million in European funding for a Phase II clinical trial for Vamorolone – also known as VBP15 – an anti-inflammatory drug …
DRF’s Chief Scientific Advisor first to correct Duchenne genetic error with cutting edge CRISPR technology
Dr Ronald Cohn, who leads the Duchenne Research Fund’s Scientific Advisory Board, has become the first ever researcher to successfully correct one of the mutations that causes Duchenne muscular dystrophy, working on cells in his laboratory at the Centre for Genetic Medicine at Toronto’s Hospital for Sick Children …
Dog with dystrophin mutation saved from Duchenne
DRF-funded research was published this week in Cell, one of the world’s most prestigious scientific journals, analysing why two golden retrievers showed no severe symptoms of Duchenne muscular dystrophy and unexpectedly lived a normal lifespan despite being born with the genetic mutation that causes Duchenne. It is now thought that the results could open up new avenues for possible treatments …
DRF’s funding of Solid GT leads to $42.5 million investment
We are thrilled that the money raised through the Duchenne Research Fund’s 2013 dinner and donations since then have enabled us to seed fund the incredible Solid GT project, which has today received industry investment (read the press release below). While gene therapy is an exciting piece of the puzzle for beating Duchenne for all boys, it is now more critical than ever that we continue to raise vital funds to push Duchenne therapies into clinical trial as quickly and safely as possible and to get them to our boys NOW.
Albourne raises £155,000 for DRF
We are immensely grateful to Albourne Partners and their supporters for raising more than £155,000 for our projects at their incredible performance of ‘The Importance of Being Lewis’ on 7 October. The event was held to honour the memory of Jacques Joubert (1992-2015) who had Duchenne …
Gene therapy breakthrough in muscular dystrophy dogs
The Duchenne Research Fund is incredibly proud of Dr Dongsheng Duan – an important Scientific Advisory Board member of Solid GT, a gene therapy programme pioneered by Solid Biosciences and that we helped to fund. Dr Duan’s latest gene therapy work on muscular dystrophy dogs is set to significantly impact 100% of the Duchenne population.
He also recently received a five-year, $3 million grant from the NIH (National Institutes of Health) …
DRF-funded study shows encouraging results in combating muscle-wasting
Professor Dame Kay Davies (University of Oxford) and her group have recently published encouraging results of a study to test new second-generation compounds to increase the level of utrophin protein as a treatment for boys and men with Duchenne and Becker muscular dystrophies.
Utrophin is a protein that occurs naturally in the body. It is usually only made in large amounts during development in the womb and is only present in small amounts in adults. Utrophin is very similar to dystrophin in its structure and previous research has shown that it can compensate for the lack of dystrophin in animal models of Duchenne muscular dystrophy …
Newcastle Plan aims to boost UK clinical trial capacity for Duchenne
Last month the Duchenne Research Fund participated in the UK National Workshop on Duchenne Muscular Dystrophy Clinical Trial Capacity at Newcastle University, hosted by TREAT-NMD. The landmark meeting brought together 75 representatives from patient organisations, pharmaceutical companies, the National Institute for Health Research and a wide range of clinicians, to discuss the challenges and solutions to increase clinical trial capacity in the UK …
Running Club triumph in 10km Vitality run
On Sunday 12 July, Ilana, Sara, Tanya, Harris, Suzanne, Corinne, Michal and Duchenne Research Fund founder Kerry (aka the Running Club) completed the Vitality British 10km London Run, raising more than £15,000 …
Charities award fellowship to vital Duchenne researcher
The Duchenne Research Fund is part of a consortium of charities working on Duchenne muscular dystrophy that has awarded a Fellowship to Dr Michela Guglieri in partnership with Newcastle University and Newcastle University Teaching Hospital to develop and advance the clinical trial agenda for this devastating and currently incurable condition. The award of £250,000 over five years is being matched by Newcastle University …
The Duchenne Research Fund partners with Solid Biosciences
With the funds we raised at our 2013 Gala Dinner we brokered the first partnership between UK Duchenne charities and commercial enterprise to launch Solid GT, an exciting gene therapy project. We approached US-based Solid Biosciences to present promising gene therapy research we had funded, and to accelerate the option of gene therapy as a treatment for Duchenne.
Leon tackles gruelling Comrades Marathon
Leon Shelley (pictured left, with fellow participant James Raanan) ran the Comrades Marathon on 31 May in South Africa – the world’s oldest and largest ultramarathon, which celebrated its 90th anniversary this year. He raised £6,500 …
David Stone wins Boroughs cup at Mini London Marathon
Last month our avid fundraiser David Stone ran the Virgin Mini London Marathon – the last three miles of the marathon. He ran in the Under 13s age group and finished his three miles in 16 minutes and 12 seconds, making him the winning runner from the London Boroughs, and sixth overall. He received his trophy live on TV from Spurs and England footballer Andros Townsend. We are so grateful to David for raising nearly £1,000 …
Reuven and Sam’s pedal power
Earlier this year Sam Millet and Reuven Cohen headed off on an epic cycle ride across Israel, raising nearly £9,000 for the Duchenne Research Fund …
Mudders tough it out to haul in £9k
On Sunday 3 May, Richard Taylor, Mike Kohn, Ilan Jacobs, Mark Preston, Neil Taub and Rich Starr braved a Tough Mudder obstacle course, raising £9,000 for the Duchenne Research Fund …
Gershon’s London Marathon run raises £6,500
We are thrilled and proud of our friend Gershon Portnoi for running the London Marathon in record time and raising an incredible £6,500 for the Duchenne Research Fund. Thank you to you and all your generous sponsors!
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