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:
10/10/2020 08:51:14 am
This isn’t true. 3 kids have died since I’ve choose to put my child in this treatment.
MTM-CNM Family Connection
10/10/2020 09:30:56 am
James, you are confusing this trial (repurposing Tamoxifen) with a completely different trial for XLMTM (gene therapy). It is true that, tragically, there have been three patients in the gene therapy trial that have passed away over the last few months and that program is currently on hold pending investigation. https://www.audentestx.com/press_release/audentes-therapeutics-provides-update-on-the-aspiro-clinical-trial-evaluating-at132-in-patients-with-x-linked-myotubular-myopathy/
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MTM-CNM Family Connection's Blog
Sharing information, resources, updates, and thoughts on various topics of interest to the MTM-CNM community.