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The CNMD Team run the Bupa London 10,000
25th May 2014

2014 Bupa 10000 team

The CNMD team with their Bupa London 10,000 medals

2014 marks the 30th anniversary of The National Brain Appeal and – for the sixth consecutive year - Professor Hanna led a Centre for Neuromuscular Diseases team, the biggest ever, with 16 Centre staff taking part.

We want to continue our vital work, and in particular we want to raise funds to support more young scientists and doctors to develop research programmes to find treatments for these serious diseases in the Centre. But to achieve this we need your help.

Any donation, no matter how small, will be gratefully received.

The Centre for Neuromuscular Diseases would truly value your support.

The dawn of translational optogenetics:
Bryson et al. restored muscle function in a mouse model of peripheral nerve injury in an anaesthetized animal
4th April 2014

Dr Barney Bryson in Professor Linda Greensmith's lab, applied tools from optogenetics in concert with ideas in regenerative medicine to restore muscle function in a mouse model of peripheral nerve injury.

The project involved a novel approach to restore muscle function following motor neuron loss, using a combination of stem cell based replacement of motor neurons and the revolutionary technique of optogenetics. Briefly, we have shown that embryonic stem cell-derived motor neurons (ESC-MNs) can be transplanted into injured peripheral nerves that have lost their endogenous motor neuron axons, and that these ESC-MNs can then reinnervate the target muscles.

Click here to view a video from Professor Linda Greensmith's lab explaining the work

Click here to view the full paper in Science

NIHR awards £450,000 for research into rare neuromuscular diseases
21 February 2014

The National Institute of Health Research (NIHR) Rare Diseases Translational Research Collaboration has awarded £450,000 to extend research into rare neuromuscular diseases. 

The award was made to the rare neuromuscular theme which is led by Professor Michael Hanna, Director of UCL’s Institute of Neurology, where research projects into Duchenne Muscular Dystrophy (DMD) and inclusion body myositis (IBM) have been funded.

Professor Francesco Muntoni, supported by the Great Ormond Street BRC, will study groups of patients with DMD and measure their particular genetic defects more precisely. 

Professor Hanna said: “The DMD programme is going to collect large cohorts nationally through the BRC infrastructure to study their natural history and their genetics accurately. We aim to understand the variation in disease severity and assess variations in treatment response in therapeutic trials."

This will be a valuable step in the translational pathway for DMD patients.

The second study will explore IBM, an inflammatory muscle disease depicted by progressive weakness and wasting of both distal and proximal muscles. Professor Hanna explained how BRC work on stratification of patients based on muscle biopsy appearances, clinical assessment and MRI evaluation will be valuable in designing and involving patients in clinical trials.

Based on the BRC’s strong links with industry it is highly likely trials of two new agents will be able to start in the next couple of years.

Arimoclomol eased inclusion body myositis in small trial

Arimoclomol showed promise as a treatment for the most common type of inflammatory myopathy in adults over age 50 in a 1-year, phase IIa, "proof-of-concept" study.

Not only was the novel oral agent "well tolerated," which was the study’s main objective to assess, but it also showed early signs that it could be effective in the treatment of patients with sporadic inclusion body myositis (IBM). Indeed, there was a trend toward slower deterioration in physical function and muscle strength for arimoclomol when compared against placebo at 8 months’ follow-up.

Click here to view the full EULAR 2013 Congress report

MRC Centre for Neuromuscular Diseases featured in new UCLH BRC Infrastructure report

Funding from our biomedical research centre (BRC) has been key to bridging
the gap between basic science and clinical outcomes for some of the most
serious muscle wasting neurological diseases such as muscular dystrophy and
peripheral neuropathies. Investment by the BRC into the MRC Centre for
Neuromuscular Diseases at UCL has enabled researchers to build up an invaluable research infrastructure, a tissue bank, patient cohorts and education and training.

See feature on page 9 of the report here.

UCLH BRC Infrastructure Report

Genomics England whole genome sequencing – an opportunity for rare disorders
Genomics England is a project set up by the Department of Health that is due to start in January 2014. The goal is to identify the genetic cause for all rare genetically undefined disorders and to sequence patients and families' genomes to help with diagnosis and the development of future treatments.

This is a very important opportunity for patients with neuromuscular disorders to have their genome sequenced in order to maximise their chance of a genetic diagnosis. This will be important for future treatments. Please click here for further information on how to have your genome sequenced.

100k Genome Project Information

Patient Information Leaflet Adult

Patient Information Leaflet 11-15 Years

Patient Information Leaflet 6-10 Years

New Sister Centre Partnership with The University of Iowa

Mike Hanna receiving his Professorship

The University of Iowa Department of Neurology is celebrating the recent expansion of its Neuromuscular Division and the new sister centre partnership with the National Hospital, Queen Square, London, with an exciting symposium focusing on the diagnosis, management, and therapies of genetic diseases of nerve and muscle. Prof Hanna, the MRC Centre Director gave a guest lecture at its inaugural event, and has been awarded the honour of Adjunct Professor in the Faculty of Neurology.

Mike Hanna receiving his Professorship with Prof George Richerson, Chairman of Neurology (left) and Prof Mike Shy, Head of the Neuromuscular Programme (right).

Click here to view the symposium programme.

Collaborative research programmes in neuropathy and MRI biomarkers have commenced between the MRC Centre and the Iowa neuromuscular programme

MRC Centenary Year 2013 - Centre Open Day
The MRC Centre for Neuromuscular Diseases celebrated the MRC's Centenary on
June 20th. The Centre was thrilled to participate in national celebrations of the MRC Centenary with a specially organised day of events for students from local schools.
Students from UCL Academy, and Channing School were invited to attend a special one day programme.

Annual Mitochondrial Patient Information Day
Saturday 6th July 2013 at Queen Square, London
Please click here to view the programme.

Muscle Study Group Annual Meeting
16-18 September 2013 at Lady Margaret Hall, Oxford

MSG 2013 Group Photo

The 2013 meeting was entitled “Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy”, and brought together members of the pharmaceutical industry, NIH and academic researchers.

The programme included four major sessions:
1. Novel Molecular Mechanisms: Implications for Therapy Development - Hereditary Myopathies
2. Overcoming Hurdles of Developing and Performing Clinical Trials
3. Novel Molecular Mechanisms: Implications for Therapy Development - Hereditary Neuropathies
4. Disorders of the NMJ: The View from Oxford

Click here to view the programme

John Morgan Hughes Memorial
Dr John Morgan-Hughes made outstanding contributions to British and world neurology as an incisive clinician, a rigorous researcher and an inspiring teacher. The John Morgan-Hughes Fellowship has been set up in his memory to support young investigators worldwide undertaking up to one year of training fellowships or other academic activities focused on mitochondrial disease research.

Please click here to view the memorial programme.