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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).

Published: Dec 20, 2017 2:20:22 PM

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.

Published: Dec 6, 2017 3:45:00 PM