The latest updates on our research projects and fundraising achievements.

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...

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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...

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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.

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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...

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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 …

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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.

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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 …

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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 …

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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 …

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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 …

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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.

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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 …

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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) …

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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 …

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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 …

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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 …

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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 …

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Our mission

We aim to stop Duchenne muscular dystrophy being a death sentence for hundreds of thousands of boys worldwide.

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Our impact

We are helping turn the hope of treatments into reality. Critical treatments are in clinical trial thanks to our funds.

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In the last 4 years we have given more than £3m to ground-breaking research projects. And there is still work to do.

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