In the Southwest, Bristol and Plymouth are two hubs of the South West Neuromuscular Operational Delivery Network (SWNODN) which provides care to over 6000 adults and children with various neuromuscular conditions. We are currently looking after people with 80 neuromuscular conditions within the network via multi-disciplinary outreach clinics. We collectively look after over a 100 boys and young men with DMD. The clinical service, which is well established and funded by NHS England, is currently highly regarded and seen as a gold standard for clinical networks within the neurology and neuromuscular community. In 2017, the SWNODN was formally recognised by Muscular Dystrophy charity, MDUK, as a Centre of Excellence for Neuromuscular conditions.
This recognition was achievable by means of a number of quality improvement activities, including guidelines, SOPs, audits about bone health and parent reported outcome measures, and feedback from parents/families questionnaire about our services. We have also established workshops and muscle cafes as a forum to provide specific information about coping, psychological support and opportunity for social networking. We have now established a specific South West DMD database to facilitate research.
Facilities we currently have within the department include:
Both of the Neuromuscular Paediatric Physiotherapists on our team are on the Neuromuscular Committee of the Association of Paediatric Chartered Physiotherapists [APCP] and have co-authored a guidance document on Physiotherapy in neuromuscular conditions produced for the APCP. Our adult Neuromuscular Physiotherapist also contributed to the adult physiotherapy e-learning module.
All of our Neuromuscular Physiotherapists are active members of the North Star network physiotherapy group and our paediatric physiotherapists contribute data to the North Star and SMArt net/SMA REACH networks
Department of Paediatric Neurology
Education Centre 6th Floor
Bristol Children's Hospital
Upper Maudlin Street
Bristol BS2 8BJ
|Study Name||Trial Status at Bristol||Overall Trial Status|
|Catabasis - Galaxy DMD [TERMINATED]||Trial complete/terminated||Trial terminated|
|Phase 3 PolarisDMD Trial [TERMINATED]||Trial complete/terminated||Trial terminated|
|Patient Registry Translarna (Ataluren)||Fully recruited||Fully recruited|
|Wave Life Sciences Exon 51||Trial complete/terminated||Trial complete|
|Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD.||Recruiting||Recruiting|
I am a Consultant in Paediatric Neurology here in Bristol. I have successfully maintained and developed a regional Paediatric Neuromuscular Service. I have introduced many of the innovations of this regional service.
I have been a major contributor to the successful business case for a Managed Clinical Network for NM conditions in the Southwest.
I have commenced and collaborated in several new research projects. I have also ensured Bristol’s participation in several national initiatives and collaborations in the Neuromuscular field (Northstar, SMArt Net/SMA REACH, TREAT- NMD). This will ensure that Neuromuscular patients in the Southwest will have access to cutting edge research and potential treatments in the future.
I am a Consultant Paediatric Neurologist in Bristol with special interest in Neuromuscular disorders. I have successfully developed local and regional paediatric neuromuscular service with outreach MDT clinics at Exeter and Torbay. As the Governance lead, I have developed may quality improvement activities including regional bone health audit, benchmarking outcomes, database, patient satisfaction questionnaire and information leaflets about steroid regimes etc.
I have commenced and collaborated in several research projects. In the Neuromuscular field, I collaborate with (North star, SMArt Net/SMA REACH, TREAT- NMD). This will ensure that neuromuscular patients in the Southwest will have access to cutting edge research and potential treatments in the future.
I am the Duchenne Muscular Dystrophy (DMD) Hub Trial Coordinator at Bristol Research and Education centre. This was a new post, funded by DMD Hub, to help provide support in the coordination of all neuromuscular related clinical trials, specifically relating to DMD. My role requires working alongside research nurses as well as the wider specialist neuromuscular multidisciplinary team. This allows me to gain a comprehensive appreciation of patients who have DMD. To begin with, I will be working on two DMD studies; Wave and Polaris. The Wave study will focus on the safety and tolerability of Exon 51 skipping therapy. While the Polaris study will evaluate the efficacy and safety of Edasolonexent in patients with DMD.
Prior to this role, I gained experience in research during my Masters in Health Psychology from University of Bath. I was involved with all the various stages of conducting research including ethics, study protocol, participant recruitment and testing, data collection, examination and dissemination. This research was part of a submitted poster presentation at the Annual Meeting of the American Psychosomatic Society conference held on March 2017 in Seville, Spain.