NewsThe latest updates on our research projects and fundraising achievements.
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…
Solid Biosciences has issued an update on IGNITE DMD, the Phase I/II clinical study with its microdystrophin gene therapy, SGT-001.read more
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…
Congratulations to the #TeamDRF cyclists who once again took on the Prudential RideLondon-Surrey, and raised more than £5,000…read more
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...read more
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 …read more
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 …
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 …read more
The Duchenne Research Fund’s co-founders Kerry and Doron Rosenfeld are honoured to have received the Prime Minister’s Points of Light award …read more
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…read more
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…read more
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...read more
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...read more
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...read more
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...read more
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.read more
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.read more
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...read more
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 …
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.read more
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.read more
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 …read more
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 …read more
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 …read more
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.read more
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 …read more
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 …read more
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 …
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 …read more
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.read more
We aim to stop Duchenne muscular dystrophy being a death sentence for hundreds of thousands of boys worldwide.
We are helping turn the hope of treatments into reality. Critical treatments are in clinical trial thanks to our funds.
In the last 4 years we have given more than £3m to ground-breaking research projects. And there is still work to do.