A 2 day international PI strategic award planning workshop was held in the MRC Centre for Neuromuscular Diseases at the UCL Institute of Neurology, on 9th and 10th October 2017. PIs from all international partner sites attended.
Published: Jan 9, 2018 12:04:58 PM
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