Individuals with MTM-CNM and their loved ones will be excited to know that there are TWO promising lines of treatment being developed for MTM: enzyme replacement therapy and gene replacement therapy!
On Saturday, July 27th, we were able to hear from two biotech companies who have invested in supporting these lines of treatment. |
 | | Hal Landy and Deb Ramsdell of Valerion hanging out with Will Ward! |
Valerion Therapeutics
In the afternoon, Deborah Ramsdell, CEO of Valerion Therapuetics, shared about the path of developing enzyme replacement therapy for Myotubular Myopathy. You can watch the video of this talk here.
Visit the Valerion website to learn more about the company. Also please take a moment to fill out this survey asking what are the top three aspects of the disease you would most like to see being improved in the treatment outcomes.
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 | | Matt Patterson having a chat with Emi Vasquez |
Audentes Therapeutics
On Saturday evening, Matthew Patterson, President and CEO of Audentes Therapeutics, spoke about his new biotech company, some fantastic fundraising news, and pursuing the path of treating MTM in the future with gene replacement therapy. Watch the video of Matt's talk here.
You can learn more about Audentes on their website. Feel free to contact Matt with any questions you might have!
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