The Children's Neurosciences department at Evelina provides a tertiary service which includes an integrated and comprehensive Neurology, Rehabilitation and Neurodisability service for children and their families from South East Thames and beyond, serving a total population of over 9 million people. We work closely with our Neurosurgical colleagues at Kings College Hospital to ensure a comprehensive Paediatric Neurosciences service is managed within Kings Health Partners.
Every year, 2500 inpatients and more than 7000 outpatients are seen within the Children's Neurosciences service. Services are provided from a recently refurbished purpose designed outpatient department in the South Wing of St Thomas' Hospital together with facilities in ELCH.
The neuromuscular service has been designated as a Neuromuscular Centre of Excellence by the charity Muscular Dystrophy UK and is one of the four neuromuscular centres in England, designated by NHS England as infusion centres for provision of the highly specialised service of delivering onasemnogene abeparvovec gene therapy to babies and children with spinal muscular atrophy. The service operates to a multidisciplinary model providing a comprehensive service for children with neuromuscular disorders, including other advanced therapies such as intrathecal nusinersen and risdiplam. The department has a strong research record, is involved in clinical trial programmes and all team members are encouraged to contribute to existing and new projects.
We have conducted several clinical trials for neuromuscular conditions, including Phase 1 studies in DMD. Dr Vasantha Gowda has experience leading clinical trials as PI and CI for the studies. In the past, we have enrolled not only our own patients but also patients from other centres in our clinical trials. We have a full-fledged ATMP set up at our centre and have treated nearly 70 patients with Zolgensma gene therapy for SMA and have clinical trial capacity for gene therapies in DMD.
We actively contribute to DMD Care and SMA Care projects and are a part of the Steering Committee for SMAREACH. We actively contribute to DMD Care and SMA Care projects and are a part of the Steering Committee for SMAREACH.
Paediatric Neuromuscular Department,
Paediatric Neurosciences,
Level 2, Becket House,
Evelina London Children’s Hospital,
Westminster Bridge Road,
London SE1 7EH
Study Name | Trial Status at London - Evelina | Overall Trial Status |
---|---|---|
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 |
Dr Vasantha Gowda is a Consultant Paediatric with neuromuscular expertise based at Evelina London Children’s Hospital, which is one of the four centres designated by NHSE for gene therapy treatment for children with Spinal Muscular Atrophy. She has an honorary appointment as a Senior Clinical Lecturer at the Faculty of Life Sciences and Medicine, King’s College.
She has served as the lead for Post-graduate medical education for paediatric neurosciences for 5 years. She has organized well attended paediatric neuromuscular study days for paediatricians and allied health professionals. With a back ground of Masters in Evidence Based Health-Care from the University of Oxford, she takes a keen interest in clinical research and serves as the co-lead for neurosciences clinical trials at Evelina London Children’s Hospital. She has successfully conducted several clinical trials as the principal/chief investigator. She is a part of the SMAREACH steering committee, which oversees data collection and monitoring for patients with Spinal Muscular Atrophy in the UK. She is a member of the Cellular, Genomic and Regenerative Medicine Board, ATMP Working Group, and the Research and Development Biological Safety Committee at the Guy’s and St. Thomas’ NHS Foundation Trust. She serves as the Regional Lead for the Royal College for Paediatric and Child Health.
Heinz Jungbluth is Professor of Paediatric Neurology at King’s College and a Consultant Paediatric Neurologist at the Evelina Children’s Hospital, Guy’s & St Thomas’ NHS Foundation Trust, London. He runs specialist clinics for children and adolescents affected by neuromuscular and neurological disorders and has more than 20 years of clinical experience in the neuromuscular field. His main research interest is in neurogenetics, in particular the genetics of early-onset neuromuscular and neurodevelopmental disorders. He has published more than 150 peer-reviewed papers and has authored several chapters in key neurological and neuromuscular textbooks. He is a member of various editorial boards, as well as National and International Expert Consortia, concerned with improving the care and developing therapies for patients affected by neuromuscular disease.
Jennie is a chartered and state-registered physiotherapist with many years of experience in paediatrics. She has worked for most of her career in paediatric neuromuscular disorders, working at a number of specialist centres, including The Hammersmith Hospital with Professor Dubowitz, then Professor Muntoni. When working at Guys Hospital, she was involved in both the Paediatric and Adult Neuromuscular clinics. For the last 11 years, she has been at The Evelina London Children's Hospital. She also has experience as a Paediatric Community Physiotherapist, and is therefore very aware of the challenges that are met by therapists working in different environments.
She works as part of the MDT team at The Evelina London, carrying out standardised assessments, providing clinical management and advice to families and local physiotherapists. She also works closely with the orthopaedic and respiratory physiotherapy teams, and the orthotists.
Jennie was one of the developers of the NorthStar Ambulatory Assessment for DMD, which after publication, became widely recognised and adopted as the standardised assessment of choice for many research studies in DMD, both nationally and internationally, and is also an essential part of the North Star national database. She is the physiotherapy representative on the National North Star Steering Committee.
Jennie has been involved in a number of research projects and publications - the most recent being the national study on hydrotherapy in DMD, as the independent assessor of prescription suitability and compliance. She has set-up, and continued to run, the South East Physiotherapy Network for NMD. She is a member of APCP and the APCP Neuromuscular group, and was previously a Committee member.
Jennie is the lead for a small team of specialist physiotherapists in Neuromuscular Disorders at Evelina London.