CMT Research Foundation

Earlier this week, Anna Combes, CFRE and Susan Ruediger visited Columbus, Ohio, for CMTRF's Ohio, What A Night! committee kick off meeting. While there, they had the opportunity to tour and see first-hand where CMT research is being done at both Nationwide Children's Hospital and The Ohio State University.   Ohio, What A Night! will help to educate and inspire businesses, biotechs and the greater Columbus community on the incredible life changing research that is happening in CMT and Gene Therapy, all while raising money for the CMT Research Foundation!   A big thank you to our committee co-chairs Michael Triplett and Nanette Nardi Triplett and our committee members Afrooz Rashnonejad, MSc, PhD, Michele and Kent Stahl, Rachel Salzman, DVM, Scott Q Harper, Lindsay Wallace, Zarife Sahenk, W. David Arnold, Kenny Morand, Laura MacNeill, M.B.A. and Cleary Simpson.    Event details will be announced soon. If you’re interested in sponsorship opportunities, please contact Anna Combes at anna.combes@cmtrf.org.

As part of country music superstar Alan Jackson’s 2024-2025 LAST CALL: ONE MORE FOR THE ROAD TOUR, CMTRF Board Chair Peter de Silva hosted a gathering of nearly 50 guests, including 5 CMT patient families, for cocktails and hors d'oeuvres preceding the concert in Kansas City on Saturday, October 26. In a private room at the T-Mobile Center, Peter, Cleary Simpson (Board Vice Chair) and Laura MacNeill (CMTRF CEO) all made short remarks about the progress CMTRF has made towards finding a cure. The group then made its way to specially selected seats in the concert for an hour and a half of Alan Jackson's greatest hits! For more information about the Tour, or to donate, please visit https://lnkd.in/djYsSrd4

CMTRF is now accepting letters of intent for the Young Researchers Innovation Grant. The YRIG supports early-career scientists developing high-impact projects to advance Charcot-Marie-Tooth disease drug development. The program provides funding for small, innovative projects, aimed at tackling key barriers in CMT research. YRIG also encourages the generation of preliminary data that can lay the groundwork for larger efforts to optimize and support therapeutic development. Collaborative projects between research groups, and between academic and industry investigators, are highly encouraged. Prospective applicants are highly encouraged to submit a Letter of Intent, a 2-3 page document outlining the idea for the project, to gain early feedback from CMTRF staff and the grant review committee that can then be incorporated into the full proposal. The LOI submission portal is open until December 13, 2024. Full applications will be accepted starting December 9, 2024 and closed on February 28, 2025. For details and guidelines on the Young Researchers Innovation Grant and LOI requirements, visit https://lnkd.in/gdFxywA7

The CMT Research Foundation is thrilled to welcome Laura MacNeill as our new Chief Executive Officer. Laura steps into the role following Cleary Simpson’s successful tenure as CEO, during which CMTRF made tremendous progress in research funding and organizational growth. Cleary will remain closely involved in our work, transitioning to the role of Vice Chair of the Board. With over 15 years of experience as a senior nonprofit executive responsible for strategic planning, innovative marketing, fundraising and raising public awareness, Laura’s expertise will be essential in advancing CMTRF’s mission. We are excited for this new chapter and look forward to Laura’s leadership as we continue advancing our mission to develop treatments and find a cure for CMT! Read more here: https://lnkd.in/gr-2gamw

This episode of Beyond the Diagnosis delves into the extraordinary journey of John Neville, a CMT patient who has defied expectations and inspired countless others. Join us as John shares his personal experiences living with CMT and the unique challenges he has faced. From navigating his diagnosis to overcoming physical limitations, John's story is one of resilience and determination. Discover how John has adapted to the challenges of CMT and found ways to maintain a fulfilling life. Hear about his personal triumphs, the support he's received from the CMT community, and the hope he finds in the future of research. To learn more about CMTRF, or to donate to help find a cure for CMT, please visit www.cmtrf.org. https://lnkd.in/gHduDKA8

Huge thank you to this year's sponsors who made the 2024 Global CMT Research Convention possible! Novartis, NMD Pharma A/S, Applied Therapeutics, Inc., Armatus Bio, Inc., Muscular Dystrophy Association, ReviR Therapeutics, Samsara Therapeutics, Actio Biosciences, Inc., Alesta Therapeutics, and Augustine Therapeutics. We hope you will join us at next year's convention—September 25-27, 2025 at the Royal Sonesta in Cambridge, MA!

The CMT Research Foundation announced over the weekend at its annual Global CMT Research Convention that it has surpassed its goal of raising $10 million to fund research to find treatments and cures for CMT Type 1A! “For the first time in more than 45 years, I am encouraged that the science is at a point where treatments and cures are soon to be a reality. We are closer than ever to finding treatments and cures. I can’t wait for that day. Because we believe that the end of CMT1A is in sight, CMTRF calls the campaign ENDGAME. Moreover, CMTRF is confident that successes achieved by the research underwritten by ENDGAME will eventually benefit all types of CMT.” - Peter J. de Silva, CMTRF Board Chair, CMT patient and Chair of the ENDGAME Capital Campaign. To read more, visit https://lnkd.in/dd3RPDs6

End clap for the CMTRF Global Research Convention in Boston. Delighted to have participated and congratulations to the entire CMTRF team and all the speakers for these 3 extraordinary days. So much progress in recent years and great prospects with the clinical trials in progress and those to come. Our communities of patients, researchers, startups and pharmaceutical industries are hard at work so that the first treatments finally arrive! CMT-France CMT Research Foundation European CMT Federation Alliance maladies rares EURORDIS-Rare Diseases Europe AFM-Téléthon

And that's a wrap on the 2024 Global CMT Research Convention!    Patient Day kicked off with US Paralympian and CMTRF Ambassador Jamal Hill as he gave a motivating speech on becoming an UNSTOPPABLE force against CMT.    The day continued highlighting CMT research. Dr. Rob Prior discussed the state of Drug Development for CMT and the exponential growth of CMT research in the last few years. Dr. Toby Ferguson provided insights from other neuromuscular disorders on how to use creative approaches to improve and accelerate clinical trials for rare diseases, like CMT. Lastly, Dr. Bruce Carter gave a comprehensive presentation explaining the genetic and biological components of CMT and how they lead to symptoms.   The day wrapped up with four CMTRF UNSTOPPABLE ambassadors—Lily Sander, Jay Mulrooney, Gary Berg and John Neville—who spoke about how they are UNSTOPPABLE and how you can be too!    We hope you will join us next year, September 25-27, 2025 at the Royal Sonesta in Cambridge, MA!

Today is Patient Day at the fourth annual Global CMT Research Convention. JOIN NOW: https://lnkd.in/dxyP_rfR More than 250 people will attend in person or virtually to connect with patients, researchers and clinicians, and learn about the latest developments in drug research. Don’t miss your opportunity to engage with other CMT patients and experience the power of community participation in driving CMT research forward at the fastest pace in history.