Steering Committee
The committee is responsible for reviewing all requests for data from the registry.
List of Steering Committee members
Dr Michela Guglieri (Chairperson)
Dr Guglieri is a Senior Clinical Lecturer and Neuromuscular Consultant at Newcastle University. She leads the clinical research team which is currently involved in over 30 studies over the past 5 years. Duchenne UK collaborated with 5 patient organisations to award a 5-year lectureship to Dr Guglieri in 2015.
Dr Anne-Marie Childs
Dr Childs has been working as a Consultant Paediatric Neurologist in Leeds since 2001 when she became the Lead of Children’s Neuromuscular Services. She is committed to delivering high-quality care to all patients with neuromuscular disorders in Yorkshire. Her clinical research interests include MR imaging and rare disease phenotyping, although her predominant research involvement is now in relation to DMD and SMA.
Emma Heslop
Emma Heslop is the DMD Hub manager and is funded by Duchenne UK. She has been part of the John Walton Muscular Dystrophy Research Centre at Newcastle University since October 2006, when she joined the TREAT-NMD Neuromuscular network of excellence.
Alex Johnson
Alex is the co-founder of Duchenne UK.
Alex founded the charity Joining Jack with her husband, ex-rugby league player Andy Johnson, following their son Jack’s diagnosis with Duchenne muscular dystrophy in 2011. The rugby community came together to help the family and helped them create international awareness for Duchenne with innovative campaigns like #Link4DMD. Alex has subsequently joined the board of United Parent Project (UPPMD) and has helped organise international awareness events like World Duchenne Awareness Day. She has represented the Duchenne community by speaking at international conferences around the world, workshops and meetings with the FDA, EMA, MHRA, NICE and at the Houses of Parliament. Alex was honoured to join the steering committee that helped organise a workshop with the EMA on exon skipping. Following the meeting the steering committee published an article in the Lancet. Alex completed training at a Eurordis summer school to become a trained patient advocate and is also a member of the MHRA patient group consultative forum
Dr Mariacristina Scoto
Originally from Italy, Dr Mariacristina Scoto is a Consultant in Neuromuscular Translational Research at Great Ormond Street Hospital for Children and is Honorary Associate Professor at UCL Great Ormond Street Institute of Child Health, London. She is principal investigator (PI) and co-PI of a number of clinical trials, in both Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). Her main interest is on the evolving natural history and standard of care of spinal muscular atrophy with the advent of innovative treatments, and is Co-PI of the SMA REACH (REsearch And Clinical Hub) UK Network.
Sejal Thakrar
Sejal has a son, Shiv, with Duchenne and they live in North West London. Shiv was diagnosed in April 2014. Following diagnosis, Sejal gave up her career as a Customer Intelligence Data Analyst within the Telecommunications sector.
In January 2015, along with her husband Manoj, they set up ‘Smile with Shiv’ to work closely with the Duchenne community, in raising awareness and funds for Duchenne charities, as well as advocacy work.