Global CMT Research Convention on Tap for Sept 16–17 in Cambridge, MA

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by Mary Chapman |

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Treatments for all types of Charcot-Marie-Tooth disease (CMT) will be the focus of the second annual Global CMT Research Convention, Sept. 16 and 17, in Cambridge, Massachusetts.

Presented by the CMT Research Foundation, the event brings together patients, scientists, and biopharmaceutical companies to celebrate treatment advancements and the pioneers who set the stage for continued progress toward a cure. The convention, which can be attended in person or virtually, will also highlight the biotechnology infrastructure in the Boston region.

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The inaugural convention last year was attended by 160 researchers from 20 countries, in addition to 300 CMT patients from 34 countries.

To open this year’s convention, the keynote speaker will be Christopher Austin, MD, CEO of Vesalius Therapeutics, and CEO-partner of Flagship Pioneering. Austin, who is a clinician and geneticist with more than 20 years of experience in the public and private sectors, is also founding director of the National Center for Advancing Translational Sciences.

Other speakers include Vincent Timmerman, PhD, who discovered the gene whose mutations cause CMT1A (called PMP22) in 1991, as well as James Lupski, MD, PhD, who made the same discovery around the same time.

“Friday, September 16th is a day for researchers to share the latest in CMT drug development, establish new connections and collaborations, and discuss pathways to treatments,” Cleary Simpson, the foundation’s CEO, said in a press release.

The following day is focused more on patients and their families. “We will give special attention to clinical trials, how they operate, why to participate in them, and when to expect them,” Simpson said. “It is important for researchers and patients to interact directly, and we are pleased to provide that forum.”

In addition to recent developments in CMT research, the Sept. 16 agenda will include presentations about metabolism and nerve fiber degeneration, genetic medicine, small-molecule approaches, and using biomarkers to de-risk clinical trials.

Keith Fargo, PhD, the foundation’s chief scientific officer, will open the Saturday session with a discussion about the history of the science of CMT and how it’s led to the current momentum in treatment development.

There will also be presentations about natural history studies and their importance, genes and their impact on treatments and prospective cures, understanding clinical trials and their design, an overview of current and upcoming clinical research, increased biotech interest, and how the patient community can influence the future of CMT.

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Breakout sessions will cover several different types of CMT, as well as pediatric issues. In addition, there will be an opportunity to network with convention speakers, followed by a sunset riverboat cruise on the Charles River.

“Once again, academic researchers, industry partners, regulatory agencies, and patients are uniting to attack key problems in drug development for CMT,” said Fargo. “This is truly an extraordinary opportunity to network with the people most critical to finding a cure for the three million people around the world with CMT.”

The convention is funded in part by DTx and Armatus Bio.

Tickets for in-person attendance are $40 for patients. There’s no charge to view the convention online.