Today (26.10.2017) is this year’s research grant from the Myotubular Trust and ZNM – Zusammen Stark! e.V. officially been awarded:
Jim Dowling of the Sick Kids Hospital in Toronto receives € 244,000 to investigate the efficacy of several promising drugs that could greatly reduce the muscle weakness of patients with myotubular myopathy.
Jim Dowling and his team have shown that in myotubular myopathy, the regulation of certain components in the walls of the muscle cells (so-called membrane lipids) is disturbed. In a second step, it was shown that the (genetic) blocking of another enzyme called PIK3C2B leads to healing in diseased mice.
Now, Jim Dowling and his team have used zebrafish to identify drugs that could effectively and safely block PIK3C2B in humans. With the research grant of the Myotubular Trust and ZNM – Zusammen Stark! Jim Dowling and his team will be able to test these drugs alone and in combination with mice over three years. The process is described in more detail in our Newsletter 2017.
Of the total cost of € 244,000, € 25,000 (over 10%) were contributed directly from ZNM-Zusammen Stark! e. V. Many thanks to all our donors who made this possible! #TogetherEvenStronger