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Orphazyme assumes sponsorship of Phase II/III arimoclomol sIBM trial

On December 14, 2017, Orphazyme A/S, University of Kansas Medical Center, and UCL announced that Orphazyme formally assumes the sponsorship of the Phase II/III arimoclomol trial for the neuromuscular disease sporadic Inclusion Body Myositis (sIBM).

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Collaborative work with Dumonceaux and Voit in Nature Communications has important implications for myostatin therapies

Muscular dystrophies are characterized by weakness and wasting of skeletal muscle tissues. Several drugs targeting the myostatin pathway have been used in clinical trials to increase muscle mass and function but most showed limited efficacy. Here we show that the expression of components of the myostatin signaling pathway is downregulated in muscle wasting or atrophying diseases, with a decrease of myostatin and activin receptor, and an increase of the myostatin antagonist, follistatin. We also provide in vivo evidence in the congenital myotubular myopathy mouse model (knock-out for the myotubularin coding gene Mtm1) that a down-regulated myostatin pathway can be reactivated by correcting the underlying gene defect. Our data may explain the poor clinical efficacy of anti-myostatin approaches in several of the clinical studies and the apparent contradictory results in mice regarding the efficacy of anti-myostatin approaches and may inform patient selection and stratification for future trials.

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Tracking disease progression in neuromuscular diseases with MRI

An Institute of Neurology study published in Lancet Neurology this week provides important prospective evidence that quantitative MRI can reliably and sensitively track disease progression in Neuromuscular Diseases. The research shows that the MRI biomarkers developed by the MRC Centre team have real potential to become the widely adopted surrogate endpoint in neuromuscular trials.

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NMCCC report

During the Bob and June Prickett Churchill fellowship in 2014, Kathryn Munro visited the NMCCC. She said of the centre:

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NIHR awards £450,000 for rare neuromuscular diseases

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

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Arimoclomol eases IBM 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.

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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.

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