Great Ormond Street Hospital Clinical Trial Co-ordinator
Manju AgarwalAs part of the Newcastle Plan in 2015/16, eight patient organisations contributed £1.2m to fund at least 16 vital posts across the UK to support and expand clinical trial capacity for Duchenne muscular dystrophy. One of those posts was a new clinical trial co-ordinator role at Great Ormond Street Hospital, which the Duchenne Research Fund, Duchenne UK and Harrison’s Fund co-funded together with Great Ormond Street Hospital Children’s Charity. Ms Manju Agarwal was subsequently recruited to fill the role. The initiative evolved into the DMD Hub, spearheaded by Duchenne UK and supported by the other charities, which aims to respond to clinical trial capacity needs across the UK. The objective of the Hub is to enable all patients to have access to clinical trials.
Clinical support post
£30,000 contribution
Funded in 2017-20
London, UK
Latest research news
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 …
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…
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 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…
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.
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...
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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