Great Ormond Street Hospital Clinical Trial Co-ordinatorManju 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.
Clinical support post
Funded in 2017-20
News from the Hospital for Sick Kids, Toronto
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 …
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...
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...
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...
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.
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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