Newcastle University has an excellent pedigree in research and teaching with a strong interdisciplinary research base. In the UK, it ranks in the top 5 for both hospital- and laboratory-based clinical subjects, with two-thirds of the outputs classified as world leading or internationally excellent in the areas of ageing, chronic disease, genetics and stem cells.
The Newcastle upon Tyne Hospitals NHS Foundation Trust (NuTH) is one of the most successful teaching NHS Trusts in the country. In partnership with the University and NuTH, the John Walton Muscular Dystrophy Research Centre is a clinical and research reference centre that is led by clinically active professors providing a depth of clinical expertise unparalleled elsewhere in the UK.
In addition to housing the Regional Diagnostic Service for Neuromuscular diseases for the Northern region, the Centre is the base for the Highly Specialised Service for Limb-girdle muscular dystrophies and attracts both clinical and laboratory referrals from the UK and beyond. The clinical team based at the centre runs multidisciplinary paediatric and adult neuromuscular clinics for over 1200 neuromuscular patients annually throughout the North of England. Clinics are held both in Newcastle and at a range of other locations throughout the region, in conjunction with local physiotherapists and other local staff. The centre has collaborative links with colleagues in cardiology, respiratory support, orthopaedics, neuropathology and other specialities.
Thanks to the strong links with the research team, the JWMDRC has been actively involved in delivering novel treatments in, clinic bringing together clinicians across different disciplines at NuTH. The Centre welcomes visiting doctors and other staff for specialised training in neuromuscular disease diagnosis and management.
The John Walton Muscular Dystrophy Research Centre (JWMDRC) is a partnership between Newcastle University and The Newcastle upon Tyne Hospitals NHS Foundation Trust Hospitals. The Centre brings together and consolidates Newcastle’s distinguished, international and world-leading record in research and care for neuromuscular diseases.
Our team of 80+ people based at Newcastle University, and its associated hospitals, work together towards the development and application of genomic and translational medicine to improve the health outcomes of people living with neuromuscular diseases.
As a group, we have developed a close and important link between research and clinical activities, and we actively pursue new partnerships with other stakeholders such as patient organisations, regulators and pharma.
The John Walton Muscular Dystrophy Research Centre is structured around five important strands of activity – clinical care, clinical research, diagnostics, basic research and strategic partnerships and networking – but all are strongly interlinked and the work across these teams is key to our success and impact in the neuromuscular field.
The John Walton Centre (JWMDRC) at Newcastle University is highly active internationally in rare disease and neuromuscular networking and healthcare policy initiatives.
The JWMDRC is the coordinating centre of the TREAT-NMD Alliance. TREAT-NMD is a network of world-leading experts for the neuromuscular field focused on the development of translational research in rare neuromuscular diseases. The network provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007, initially as a European Union funded Network of Excellence, the focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.
TREAT-NMD is committed to promoting collaborations between all stakeholders in order to avoid fragmentation and duplication. Its members include clinicians, patients, industry, academic institutions, advocacy groups and regulatory agencies, all of whom recognize the advantages of strong interactions in accelerating cutting-edge therapies for otherwise unmet medical needs.
Since 2012 the JWMDRC has coordinated the EUCERD Joint Action on Rare Diseases, responsible for aiding the European Commission with the preparation and implementation of Community activities in the field of rare diseases. Staff have also been in key coordinating roles in other European projects, including RD-Connect, BIO-NMD, CARE-NMD, NMD-Chip and EuroBioBank, and have been responsible for scientific research into muscular dystrophies in EU research projects such as Neuromics.
John Walton Muscular Dystrophy Research Centre
Translational and Clinical Research Institute
International Centre for Life
Newcastle upon Tyne
|Study Name||Trial Status at Newcastle||Overall Trial Status|
|Sarepta - EMBARK||Fully recruited||Fully recruited|
|Pilot Study Comparing night time AFOs with CCDs in the management of ankle contractures||Fully recruited||Fully recruited|
|Genethon - Natural History of Duchenne Muscular Dystrophy||Recruiting||Recruiting|
|D3 Creatine||Enrolling by invitation||Enrolling by invitation|
|Pfizer - CIFFREO||Recruiting||Recruiting|
|Italfarmaco - Givinostat Extension||Fully recruited||Fully recruited|
|Testosterone in DMD follow up study||Trial complete/terminated||Trial complete|
|Sarepta Extension Study for Casimersen or Golodirsen||Enrolling by invitation||Enrolling by invitation|
|Santhera SIDEROS Open Label Extension [TERMINATED]||Trial complete/terminated||Trial terminated|
|DYSTANCE 51 [TERMINATED]||Trial complete/terminated||Trial terminated|
|Patient Registry Translarna (Ataluren)||Fully recruited||Fully recruited|
|Vamorolone Phase 2b (VISION-DMD)||Trial complete/terminated||Trial complete|
|Santhera (SIDEROS) [TERMINATED]||Trial complete/terminated||Trial terminated|
|Sarepta 53||Trial complete/terminated||Trial complete|
|Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD.||Recruiting||Recruiting|
|Brain study||Trial complete/terminated||Trial complete|
|PTC Ataluren Phase 3||Trial complete/terminated||Trial complete|
|Ataluren long-term||Trial complete/terminated||Trial complete|
|Vamorolone Phase II Extension||Trial complete/terminated||Trial complete|
|Sarepta- ESSENCE||Fully recruited||Fully recruited|
|Italfarmaco- Givinostat (EPIDYS)||Trial complete/terminated||Trial complete|
|FOR-DMD||Trial complete/terminated||Trial complete|
|Outcome measures||Trial complete/terminated||Trial complete|
|Testosterone for DMD||Trial complete/terminated||Trial complete|
I was founding joint co-ordinator of TREAT-NMD, and am an executive board member of the World Muscle Society and R&D Director of the North Tees and Hartlepool NHS Foundation Trust. Together with Hanns Lochmüller, I was responsible for setting up the German National Muscular Dystrophy Network, MD-NET, of which I was joint coordinator until 2008.
I trained as a paediatric neurologist at the Universities of Düsseldorf and Essen in Germany. After completing my PhD thesis on Duchenne muscular dystrophy, I worked as a Postdoctoral Research Fellow in the laboratory of Kevin Campbell at the University of Iowa. In 2003, I joined Newcastle University's Institute of Genetic Medicine as the Professor of Neuromuscular Genetics.
I have a long-standing interest in the pathogenesis of muscular dystrophies and within the Neuromuscular Research Group at Newcastle, am engaged in research using zebrafish and mouse models. I have a special interest in the use of MRI in muscular dystrophies and the insight that contrast-enhanced MRI and quantitative MRI can give into the dynamic processes underlying muscle fibre de- and regeneration.
I did my basic medical and specialist training in Adult Neurology in Milan, Italy.
Since 2007, I have been working with the muscle team in Newcastle with a specific interest in inherited neuromuscular diseases. I have a particular interest in clinical research and clinical trials, and since 2008, I have been involved in several different trials in Duchenne Muscular Dystrophy and other neuromuscular disorders.
I am currently Research Fellow and Honorary Consultant, as well as study chair for VISION-DMD
Professor Jordi Díaz-Manera joined Newcastle University in 2020 where he is working as Professor of Neuromuscular Disorders and as Honorary Consultant Clinical Geneticist with the Newcastle Hospitals NHS Foundation Trust.
Jordi has a long-standing experience in translational clinical and basic research. His current clinical research projects are focused on the characterization of large cohort of patients with muscular dystrophies, the implementation of quantitative muscle MRI for the diagnosis and follow-up of patients with muscular diseases and the development of new outcome measures for clinical trials and natural history studies.
His basic research interest focuses on the process of muscle fiber degeneration and the replacement of muscle tissue by fat and fibrosis that takes place in patients with muscular dystrophies. He is especially interested in translating outcomes from cell biology experiments to the development of new therapies, and test them in preclinical animal models of muscular dystrophies.
I am a Consultant Research Physiotherapist, particularly interested in the development of outcome measures for the clinical assessment of neuromuscular diseases and specifically the evaluation of functional scales by Rasch analysis. I am one of the international trainers for the GSK/Prosensa programme, and am involved in the SMARTNET and North Star projects for SMA and DMD. I also conduct clinical evaluation for patients and am involved in clinical trials.
Meredith James is a Clinical Specialist Neuromuscular Physiotherapist at the John Walton Centre for Muscular Dystrophy Research at Newcastle, where she is involved in clinical and research activity for children and adults with Neuromuscular disorders.
Meredith moved to the UK in 2004 from Australia and worked in a variety of locations before joining the Oswestry Muscle team in 2006 and coming to Newcastle in 2012.
Clinically, Meredith is responsible for the physiotherapy management of both children and adults with neuromuscular diseases. In particular, her interests are in assessment, orthotics, outcome measures and research into these areas.
In her clinical research capacity, Meredith is responsible for the clinical evaluation of children and adults involved in natural history and clinical trials, as well as the development of clinically meaningful, reliable and sensitive outcome measures for NMD.
I am a Neuromuscular Physiotherapist at the John Walton Muscular Dystrophy Research Centre in Newcastle upon Tyne (UK). I started working with neuromuscular patients back in Barcelona in Hospital Sant Joan de Déu as a Paediatric Physiotherapist. In 2014, I moved to the JWMDRC to work exclusively in NMD. My professional activity covers provision of clinical management and research activity in Neuromuscular diseases with a special interest in SMA. Robert is currently undertaking a PhD.
I am a neuromuscular research physiotherapist based at the John Walton Muscular Dystrophy Centre. I joined the team in March 2017. Prior to my current role I have worked as a senior physiotherapist working with people with learning and developmental disabilities.
My current role allows me to work across both clinical and research strands. I am currently involved with multiple clinical trials in Duchenne muscular dystrophy, as well as a natural history study in dyferlinopathy.
I am a physiotherapist and have been qualified for 15 years. My background is neuro-rehabilitation and I joined the Newcastle Muscle team is 2016. I work with both children and adults with neuromuscular disease in clinical trials. I am currently studying for a Masters in Clinical Research at Newcastle University.
I am a paediatric research nurse and I have worked on the CRF since 2012 delivering early phase clinical trials to both children and adults. The disease areas I have worked with on clinical trials have included DMD, SMA, Achondroplasia, Haemophilia and JIA. Other clinical trials in set up are looking at epilepsy, mitochondrial disease and autism treatments. We enrol participants regionally, nationally and internationally.
We have a dedicated paediatric area within the department and aim to deliver family centred, holistic care working with both academic and pharmaceutical partners to develop new treatments for chronic and rare diseases. We have delivered the first UK Pfizer DMD gene therapy dose in May 2021 and I am currently involved in the set-up of a number of other gene therapy studies.
I have maintained a keen interest in research since completing my MA in Psychology in 2008. Working as a Research Assistant, Assistant Psychologist and Research Associate in academic institutions and more recently acting as Industry Coordinator for the Clinical Research Network (NENC) has ensured that I have experience in delivering as well as coordinating research.
In my role as Clinical Trials Coordinator for the JWMDRC, I help to coordinate the wide portfolio of trials delivered by the team. My work spans the early stages of developing and costing grant applications and assessing study feasibility, through to formal study set-up activities and then monitoring study delivery. All aspects of my role involve adhering to Good Clinical Practice and ensuring that research is conducted in accordance with UK regulations and local guidance. I work collaboratively with teams within Newcastle University, the Clinical Research Facility and the Newcastle-upon-Tyne Hospitals NHS Foundation Trust, together with Sponsors and Contract Research Organisations to get trials up and running as efficiently as possible.
As a Clinical Trial Coordinator in the JWMDRC, my role is to support the setup and coordination of our clinical research portfolio, including natural history studies, clinical trials of an investigational medicinal product and gene replacement therapy. I support the early stages of research, from grant applications for new research proposals and site feasibility, to the set-up and delivery of world-class research. I am part of a wider collaborative team in The Newcastle upon Tyne Hospitals NHS Foundation Trust and the University of Newcastle, who work seamlessly together in order to deliver exceptional research, with the aim to better the care and treatment of those with neuromuscular conditions
As a Clinical Trials Coordinator, I am responsible for the coordination of a portfolio of clinical research studies in Genetic Neuromuscular Diseases within the JWMDRC. This includes early approvals, start-up, and maintenance and close out of clinical studies. I am part of a wider collaborative team in The Newcastle upon Tyne Hospitals NHS Foundation Trust and the University of Newcastle, who work together to deliver research activities, targeting a better care and treatment of those with neuromuscular conditions.
I am a qualified Nurse and Midwife, with experience working in different specialities but most of my career I spent as Midwife/Head Midwife in both public and private hospitals. My research experience includes data management and coordination in hepatic diseases and now in Genetic Neuromuscular Diseases.
As a Clinical Trial Coordinator at the JWMDRC, my role is to support the set-up and coordination of our clinical research portfolio, which includes clinical trials of an investigational medicinal product and gene replacement therapy.
I 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).
I work closely with teams within Newcastle University, the Clinical Research Facility and the Newcastle-upon-Tyne Hospitals NHS Foundation Trust, together with Sponsors and Contract Research Organisations across the world to get trials up and running as efficiently as possible in order to facilitate the delivery of world class research.
I am qualified dental specialist and consultant endodontist. I have completed MRes Translational medicine and therapeutics from Newcastle University covering in depth knowledge on drug discovery and experimental clinical trials in UK and EU. My key research interests are inflammation and identification of novel pain pathways. My Ph.D. project on dental pulp stem cells in Translational and Clinical Research Institute, Newcastle University made be experienced in RNA sequencing, various wet lab skills involved in transcriptomics and proteomics, data analysis and management. As a Clinical Trials Coordinator, I am responsible for the management of clinical research studies in John Walton Muscular Dystrophy Research Centre. The scope of my role is not limited to early approvals, set-up, and maintenance and closure of clinical trials.
Within my role, I support the Clinical Trial Team in the setup, maintenance and delivery of the neuromuscular research portfolio. This includes, but is not limited to data entry and management, arranging trial appointments, facilitating study specific training and working closely with the clinical team and coordinators to enable the efficient and effective delivery of trials.
Part of my role is to also support the efficient working across wider multi-disciplinary teams, including the Clinical Research Facility and The Newcastle upon Tyne Hospitals NHS Foundation Trust support teams, in order to facilitate the delivery of world class translational research.
As a collaborative research centre my role is also cross-cutting with the wider administration team, which enables all project staff to support the wider team.