First Global CMT Research Convention Planned for September

The CMT Research Foundation (CMTRF) will host the first Global Charcot-Marie-Tooth disease (CMT) Research Convention on Sept. 24–25 in Alexandria, Virginia, and online.

“The CMT scientific community is growing rapidly. We know that real progress happens in environments that foster open collaboration and sharing of science,” Susan Ruediger, co-founder and CEO of CMTRF, said in a press release.

“The CMT Research Convention is designed to facilitate greater sharing of ideas, identify areas of need and opportunity for high-impact collaborative research projects, and to update patients on our remarkable progress in just three years,” Ruediger added.

Speakers will include researchers from international insitutions, as well as members of patient advocacy organizations, such as the Muscular Dystrophy Association.

“The 2021 Global CMT Research Convention unites patients, researchers, pharmaceutical companies, and government agencies to streamline efforts, collaborate more effectively, and to accelerate strategies for CMT treatments,” said Keith N. Fargo, PhD, CMTRF’s chief scientific officer.

“We expect,” he continued, “hundreds of researchers and pharma industry leaders both in-person and joining virtually from around the world, to work together to identify and solve key barriers to drug development for CMT.”

The convention features a full-day meeting for the research community on Sept. 24. The next day patients will have the opportunity to interact with researchers, allowing them the opportunity to discuss ongoing research and its challenges, how to become involved, and more.

“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,” Fargo said.

Arthur T. Suckow, PhD, CEO of DTx Pharma, and James M. Wilson, a gene therapy innovator from the University of Pennsylvania, will deliver keynote addresses.

DTx, a co-sponsor of the convention, is currently researching several ways to use RNA molecules to treat the underlying defect driving CMT type 1A.

Wilson has developed virus-based gene therapy candidate for genetic disorders such as CMT2A.

Other speakers include: gene therapy researchers Zarife Sahenk and Kleopas Kleopa; nerve health experts Jeff Milbrandt and Ludo Van Den Bosch; and Rob Burgess and Cat Lutz, experts in designing animal models of disease.

The convention will be streamed online to a global audience.

“The CMT Research Foundation has one single focus: deliver treatments and cures for CMT during our lifetime,” said Patrick Livney, co-founder and Chairman of the CMTRF.

“We are a patient-centered foundation, united in one cause: ENDING CMT,” he said. “It is critical that patients be involved in the drug development process — from lending their voices to discovering important outcomes to participating in clinical trials. Patients are why we exist and are at the heart of everything we do.”