MDUK Oxford Neuromuscular Centre, University of Oxford

  • 13-Nov-2019

The MDUK Oxford Neuromuscular Centre ( was initiated through a partnership between Muscular Dystrophy UK and the University of Oxford. A 5-year Centre funding award from the charity and a significant investment from the university, have laid the grouds on which we are now building a centre of excellence for neuromuscular research and clinical trials. 

The Centre, established in January 2019, is located within the University of Oxford's Department of Paediatrics, and is a broad partnership across multiple departments of pre-clinical and clinical expertise. It builds on the already excellent neuromuscular disease research, training and patient care in Oxford, to drive the development of novel experimental medicines to impact neuromuscular diseases, developing enhanced clinical trial capacity in adults and paediatrics. The Centre is governed by a steering group of researchers all based a the University of Oxford: Professors Matthew J.A. Wood, Dame Kay E. Davies, Kevin Talbot, Laurent Servais and Georg A Hollander and managed by the Centre's project manager, Dr Jaclyn Nicole le Grand. 

University of Oxford and Oxford University Hospitals NHS Foundation Trust

Department of Paediatrics, University of Oxford

The Department of Paediatrics was established in 1972 and is a part of the Medical Sciences Division.  The Department has a major interest in infectious and neuromuscular diseases on infancy and childhood and comprises clinical, teaching and research facilities within Oxford Children’s Hospital, the John Radcliffe Hospital’s Women’s Centre, the Institute of Molecular Medicine, the Peter Medawar Building, the Wellcome Trust Centre for Human Genetics (WTCHG), and the Centre for Clinical Vaccinology and Tropical Medicine (CCVTM).  The department currently employs around 160 clinical practitioners, research scientists and administrative staff and have an annual non-research turnover in excess of £6.5 million, with more than 120 active research grants.

For more information please visit:  

The University of Oxford is a member of the Athena SWAN Charter and holds an institutional Bronze Athena SWAN award. The Department of Paediatrics holds a departmental silver Athena SWAN award in recognition of its efforts to introduce organisational and cultural practices that promote gender equality and create a better working environment for both men and women.

Oxford University Hospitals NHS Foundation Trust

Oxford University Hospitals NHS Foundation Trust (OUH) is a world renowned centre of clinical excellence and one of the largest NHS teaching trusts in the UK. It became a Foundation Trust on 1 of October 2015, enabling it to work more effectively in partnership with patients and the local community to provide high quality healthcare.

Adult and Paediatric Neuromuscular Services, Oxford University Hospitals

Patients with neuromuscular diseases have access to different clinical services integrated under the idea of excellence in care for neuromuscular disorders: the Oxford Motor Neuron Centre, the nationally funded Congenital Myasthenia Service, the Adult Muscle Service, the Peripheral and Pain Service, and the nationally funded Mitochondrial Service. All our services are centred at the John Radcliffe Hospital in Oxford. All services are funded by NHS England with support of the University of Oxford, and are associated with active translational research programmes.

The Paediatric Neuromuscular Service is based at Oxford Children’s Hospital. The team provides a regional neuromuscular service for surrounding counties including Oxfordshire, Buckinghamshire, Berkshire, Northamptonshire and parts of Wiltshire, Gloucestershire and Warwickshire. The team runs a one-stop multi-disciplinary clinic for children with neuromuscular disorders and has close links with the cardiology, respiratory, orthopaedic, spinal, neuropathology and radiology services to provide a comprehensive care. There is an established joint transition clinic with the Adult Muscle Service which comprises a specialist Neuromuscular Consultant, Specialist Clinical Research Physiotherapist, Neuromuscular Care Advisor, and MDUK funded Advocacy Officer.

Experience of running trials

The paediatric and adult neurology teams perform neuromuscular clinical trials and research with support from the NIHR Clinical Research Network coordinators and research nurses.

Current trial activities include:

  • Phase 3 DMD clinical trial run by Santhera (SIDEROS) – trial open and recruiting
  • Wave exon 51 skipping- trial due to open recruitment soon
  • Patient registry Translana (Ataluren)

Past neuromuscular trials in which Oxford has participated include:

  • LVJJ
  • DMD Heart Protection

Other relevant projects

Researchers and clinicians at the MDUK Oxford Neuromuscular Centre work on a range of neuromuscular diseases including motor neuron disease, muscular dystrophies, neuromuscular junction synaptophathies, peripheral neuropathies and spinal muscular atrophy. For more information about the neuromuscular research being carried out at the Universityy of Oxford, please visit our research page at:

Through his affiliation in Belgium, Professor Servais is involved in the following trials:

  1. Wave phase 1 and 3
  2. Essence (Sarepta)
  3. Sunfish
  4. Rainbowfish
  5. Italfarmaco (Givinostat)
  6. Sideros
  7. Stive EU (Gene therapy trial Avexis)
  8. Sprint (Gene therapy trials Avexis pre-symptomatic patients)

Contact Information

Department Name
University of Oxford and Oxford University Hospitals NHS Foundation Trust
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STRONG Team Office

Level 3,

Academic Centre,

John Radcliffe,



Primary Email Address
Primary Contact Person
Patients: Laura Chiverton,
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Current Trials:

Study Name Trial Status at MDUK Oxford Neuromuscular Centre, University of Oxford Overall Trial Status
Italfarmaco - ULYSSES Not yet recruiting Recruiting
Roche - ENVOL Not yet recruiting Not yet recruiting
DMDhome Recruiting Recruiting
Sarepta ENVISION Not yet recruiting Recruiting
Sarepta - EMBARK Fully recruited Fully recruited
WVE - N531 Fully recruited Fully recruited
Sarepta - MOMENTUM Fully recruited Fully recruited
DYSTANCE 51 [TERMINATED] Trial complete/terminated Trial terminated
Santhera (SIDEROS) [TERMINATED] Trial complete/terminated Trial terminated
Sarepta- ESSENCE Fully recruited Fully recruited

Contact Listing:

Laurent Servais is a Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre and Invited Professor of Child Neurology at Liège University.

After graduating from Louvain Medical School, Brussels, Belgium in 1999, he completed a PhD in Neuroscience (cerebellar electrophysiology in alert living mice) from Free University of Brussels, Belgium, followed by residencies in child neurology at the Free University of Brussels and Robert Debré Hospital, Paris. In 2008, he took a position in neuromuscular disease and clinical research at the Institute of Myology in Paris, where his interest and expertise in neuromuscular diseases flourished. He was subsequently appointed Head of Clinical Trials and Database Services. Most recently, he served as Head of the Institute of Myology’s I-Motion (Institute Of Muscle-Oriented Translational Innovation), and Head of the Neuromuscular Centre in Liège, Belgium. He has joined the MDUK Oxford Neuromuscular Centre and the University of Oxford as of September 2019.

Laurent has been involved as principal investigator in numerous clinical trials to test treatments for Duchenne muscular dystrophy and spinal muscular atrophy (SMA).

He is the leader of the newborn screening programme for SMA in southern Belgium where they are conducting a medico-economic analysis of newborn screening. His main research expertise covers the development of innovative outcome measures, including connected devices for real-life patients’ evaluation.

+44 01865 234248

Dr Stefen Brady is a Neurologist and Specialist Neuromuscular Consultant. He studied medicine at Trinity College Medical School in Dublin, Ireland and completed his higher specialist neurology training in Yorkshire. He trained in muscle diseases with Dr David Hilton-Jones at the John Radcliffe Hospital in Oxford and with Prof. Mike Hanna and Prof. Janice Holton at MRC Centre for Neuromuscular Diseases in London. 

Dr Brady was an MDUK funded Clinical Fellow. He was awarded a DPhil by the University of Oxford for his thesis, A Clinicopathological Study of Inclusion Body Myositis (IBM), in 2014. Following his clinical fellowship, he was funded by Myositis UK to complete a Senior Clinical Research Fellowship at the MRC Centre for Neuromuscular Diseases in London.

Dr Brady has worked as a Specialist Neuromuscular Consultant since 2016. He leads the multidisciplinary Oxford Adult Muscle Service and works for the nationally commissioned NHS Highly Specialised Services for Rare Mitochondrial Disorders in Oxford.

Sithara Ramdas has been a Consultant Paediatric Neurologist at Oxford Children’s Hospital since 2016, and Honorary Senior Clinical Lecturer with the University of Oxford since 2018. She completed her higher specialist training in Paediatric Neurology and sub-specialisation in neuromuscular disorders at the King’s College Hospital and the Evelina Children’s Hospital, London.

She undertakes full time clinical practice in Paediatric Neurology. Her areas of specialist interest are in Neuromuscular Disorders and Neuroimmunology. She leads a specialist Multi-disciplinary Paediatric Neuromuscular Clinic which is the regional neuromuscular service covering Oxfordshire, Northamptonshire, Buckinghamshire, Bedfordshire and Wiltshire.  She also works in the National Highly Specialised Service for Congenital Myasthenia Syndromes and Neuromyelitis Optica.

She leads the research and clinical trials in the paediatric neurology department.


Laura Chiverton is the Clinical Trials Co-ordinator and Operations Manager for STRONG, led by Professor Laurent Servais at the MDUK Oxford Neuromuscular Centre, University of Oxford. She manages a portfolio of academic and industry led studies in the paediatric neuromuscular field.

Laura is a qualified nurse and has four years of experience working as a research nurse managing a portfolio of neuromuscular and renal research trials. Laura has recently joined STRONG and is excited to be working with such a dynamic team.

Since my Masters degree in Physiotherapy obtained in June 2013, I started to work in September 2013 as Physiotherapist at the Institute of Myology; a centre specialized in care and research in adult and paediatric neuromuscular diseases. Over the course of my career, I have participated in the creation of the I-Motion Institute, and was the Physiotherapy Coordinator for an international natural history study in Myotubular Myopathy. 

I am also a part of the ActiMyo team, composed of engineers and clinicians. This innovative device uses magneto-inertial sensors to permit movement, gait and activity analysis in uncontrolled environments, and one of its application has been qualified by the European Medical Agency as a clinical endpoint in clinical trials in Duchenne muscular dystrophy. My role is to give a physiotherapy point of view and input, and to train the physiotherapists conducting the clinical trials. Since 2018, I’m a Master trainer and consultant for scales and outcome measures used in neuromuscular clinical trials and clinics.

After more than six years of collaboration with Laurent Servais in Paris, I decided to join his team within the MDUK Oxford Neuromuscular Centre, a partnership between the University of Oxford and Muscular Dystrophy UK to drive the development of new therapies and increase national clinical trial capacity in neuromuscular diseases. .

Hayley is a Specialist Neuromuscular Physiotherapist, at Oxford University Hospitals. She sees a large cohort of patients with neuromuscular conditions, who access Oxford as their tertiary centre for neuromuscular and respiratory care.

She supports the Oxford diagnosis specific multidisciplinary clinics, which run on a weekly basis and also provides outreach support to general neurology clinics and offers community and hospital visits as required.

Within her role she supports patients in their orthopaedic and respiratory management, offering input during planned and unplanned admissions and linking in with other members of the wider multidisciplinary team and community therapists.

She is also a part of the Oxford Congenital Myasthenia Service. This is a nationally funded service, which includes both paediatric and adult patients. She is currently working on projects in collaboration with other specialist physiotherapy colleagues, on the application of outcome measures and exercise advice in this patient cohort.

As an Assistant Clinical Trial Manager at the Oxford (JR Hospital), my role is to support the set-up and coordination of our clinical research portfolio, including natural history studies & also clinical trials of an investigational medicinal product and gene replacement therapy. This includes early approvals, start-up, and maintenance and close out of clinical studies. I also support the studies at a very early stage from feasibility to costing negotiations to the set-up and monitoring delivery of research, ensuring research studies that are conducted adhere to Good Clinical Practice (GCP).

Our team called STRONG (specialised translational research oxford neuromuscular group) focuses on research into early identification, potential treatments and early intervention of rare paediatric neuromuscular diseases. As a clinical trial coordinator, I have the responsibility for the delivery of a portfolio of trials, in these areas, from set up to closeout whilst ensuring trial quality and data integrity. It is an extremely dynamic role ranging from liaising with a multidisciplinary team, interacting with patients to processing bloods alongside learning about the biological mechanisms and presentation of paediatric neuromuscular diseases.

Rebecca has been a Family Care Advisor at the John Radcliffe Hospital since July 2016, working mainly with the paediatric neuromuscular service but also with the mitochondrial team. She shares the mitochondrial FCA role with her colleague, Heather Ryan, tending to concentrate on paediatric work. She graduated as an Occupational Therapist from Northumbria University in 1992, and gained an MA in Play Therapy from the University of York in 2003. She has worked mainly in Child and Adolescent Mental Health Services (CAMHS), though she has also worked in learning and physical disabilities and in community paediatrics. Before her current role she spent 14 years as a Clinical Specialist Occupational Therapist in CAMHS in Swindon.

Gita Thapaliya is currently an Clinical Trial Manager for STRONG (specialised translational research oxford neuromuscular group) working on a portfolio of academic and industry-led studies in the paediatric neuromuscular field. Gita graduated from King’s College London in Nutrition. She then moved to the University of Nottingham to complete her PhD, followed by a postdoctoral fellowship at Johns Hopkins Medicine in the United States. Gita has worked in academic units at the University of Nottingham and Johns Hopkins conducting her own research, as well as supporting investigators to conduct high-quality clinical research in therapeutic areas including inflammatory bowel disease, obesity and ADHD.

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