Newcastle University and Newcastle upon Tyne NHS Hospital Foundation Trust

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.

Newcastle upon Tyne NHS Hospital 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 the NuTH. The centre welcomes visiting doctors and other staff for specialised training in neuromuscular disease diagnosis and management.

John Walton Muscular Dystrophy Research Centre

The John Walton Muscular Dystrophy Research Centre (JWMDRC) is a partnership between Newcastle University and Newcastle upon Tyne NHS Hospital 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.

Other relevant projects

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. http://www.treat-nmd.eu/

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.

Contact Information

Department Name
John Walton Muscular Dystrophy Research Centre
Visit Us

John Walton Muscular Dystrophy Research Centre 
Institute of Genetic Medicine
Newcastle University 
International Centre for Life 
Newcastle upon Tyne
NE1 3BZ

Primary Phone Number
+44 (0) 191 241 8610
Primary Contact Person
Patients: Katherine.Frith@newcastle.ac.uk
Industry: Anna.Peel1@newcastle.ac.uk
National Institute for Health Research (NIHR) Contact:
Steven Pratt, stevenpratt@nhs.net
 
 
View Clinical Trials At This Site  
 
 

Current Trials:

Study Name Status
Patient Registry Translarna (Ataluren) Recruiting
Vamorolone Phase 2b (VISION-DMD) Not yet recruiting
Santhera (SIDEROS) Recruiting
Sarepta 53 Ongoing (fully recruited)
Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD. Recruiting
Brain study Ongoing (fully recruited)
PTC Ataluren Phase 3 Trial complete
Ataluren long-term Trial complete
Vamorolone Phase 2 Extension Enrolling by invitation
Sarepta- ESSENCE Recruiting
Summit- Ezutromid (PhaseOut DMD) Trial terminated
Italfarmaco- Givinostat (EPIDYS) Ongoing (fully recruited)
FOR-DMD Ongoing (fully recruited)
Domagrozumab extension Trial terminated
Domagrozumab phase 2 Trial terminated
Outcome measures Ongoing (fully recruited)
Testosterone for DMD Ongoing (fully recruited)
 
 

Contact Listing:

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 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

michela.guglieri@ncl.ac.uk

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.

anna.mayhew@newcastle.ac.uk

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 now 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.

meredith.james@ncl.ac.uk

I am a Neuromuscular physiotherapist at the John Dalton 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.

0191 2418865
robert.muni-lofra@ncl.ac.uk

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 and in clinical trials. I am currently studying for a Masters in Clinical Research at Newcastle University. 

dionne.moat@newcastle.ac.uk
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.
jassi.sodhi@newcastle.ac.uk

I qualified as a paediatric nurse in March 2015, and worked on a children’s ward within the Royal Victoria Infirmary before joining the muscle team as a Research Nurse in September 2016.  My role was introduced to help increase trial capacity for patients with DMD as part of the ‘Newcastle Plan’ and funded by Duchenne UK and Joining Jack. I currently work across two sites, at the Clinical Research Facility at the Royal Victoria Infirmary and at the John Walton Muscular Dystrophy Research Centre.

The role includes the management of all aspects of managing a clinical trial, from the initial setup of a study to the day-to-day delivery of trials. My clinical skills involve venepuncture, cannulation and portacath access, as well as recording ECGs and processing samples. I develop professional relationships with the patients and families enrolled on trials and provide holistic care and support during their participation in a study. My role has allowed me to develop links with the muscle team that enables the sharing of relevant knowledge and information about the patient group as well as increasing trial capacity.

01912820070
katie.waller@newcastle.ac.uk

Since January 2016, I have worked as Project Manager for VISION-DMD, a Horizon 2020 funded drug development programme.  The Vision-DMD project will undertake Phase 2a and 2b clinical trials investigating the safety and efficacy of Vamorolone (VBP15) in ambulant Duchenne muscular dystrophy (DMD) boys.

Prior to this, I worked in a clinical research setting for 7 years as lead clinical trial coordinator at the JWMDRC during which time I gained a wide range of experience in clinical trials set up, financing and regulatory standards.  As part of the team and working with the Principal Investigators I maintain an oversight of the trials in the JWMDRC.

0191 241 8697
becky.davis@ncl.ac.uk

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.

0191 241 8649
anna.peel1@newcastle.ac.uk