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
FDA approves first gene therapy for Duchenne
The FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world …
See the smart suit that is changing children’s lives
On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart suit’ aims to help children who are losing upper body function in still continuing to use their arms.
DRF-funded KineDMD study published in Nature Medicine and featured on BBC
We are delighted that the ground-breaking KineDMD activity monitoring study we funded has been published in the prestigious scientific journal Nature Medicine, and covered by the BBC …
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