Great Ormond Street Hospital Clinical Trial Co-ordinator

Manju Agarwal

As 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.

GOSH

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 …

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

ReveraGen logo
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…

DRF-funded Imperial College AI study extended

KineDMD at Imperial
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…

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 funds Newcastle refurbishment

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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