Our "Community Conversations" series, co-hosted by MTM-CNM Family Connection and Where There's A Will There's A Cure, continued on July 13th 2020, with an excellent presentation and Q&A from Dr. Jim Dowling of the Hospital for Sick Children in Toronto, Ontario. There are several therapeutic approaches being studied for X-Linked Myotubular Myopathy, all borne out of years of hard work and research by many people, but this one also came about with a bit of "serendipity". Through an incidental and unexpected outcome in the laboratory while working with a mouse model of MTM, Dr. Dowling's team discovered that the anti-cancer drug tamoxifen seems to have a positive effect on the disease. This finding was also published by another team of researchers working completely independently. While the exact mechanism of how tamoxifen effects the disease processes in MTM is still unknown, the preclinical data from the mice is compelling. Dr. Dowling is now preparing to launch a human clinical trial to test the safety and efficacy of tamoxifen as a treatment for XLMTM. Watch the replay of the presentation (recorded live on Zoom) here:
On June 29th 2020, MTM-CNM Family Connection and Where There's a Will There's a Cure co-hosted a live Zoom presentation for the MTM-CNM family community. The guest speaker was Chris Freitag, Chief Medical Officer of Dynacure, who provided an update on the company's "UNITE-CNM" clinical trial. We were pleased to have over 40 community members tune in live for the presentation and question-and-answer session that followed. Watch the replay of the video here:
MTM-CNM Family Connection's Blog
Sharing information, resources, updates, and thoughts on various topics of interest to the MTM-CNM community.