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- The largest CNMD team to date ran the Bupa London 10,000 on 25th May to raise funds for the Centre. Please click here to donate.
- The dawn of translational optogenetics: Bryson et al. restored muscle function in a mouse model of peripheral nerve injury
- NIHR award MRC Centre for Neuromuscular Diseases £450,000 for research
- Arimoclomol has 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
- MRC Centre featured in UCLH BRC Infrastructure Report
- Genomics England whole genome sequencing
- New sister centre partnership with the University of Iowa Department of Neurology
The CNMD Team run the Bupa London 10,000
25th May 2014
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.
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.
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.
New Sister Centre Partnership with The University of Iowa
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