CMT Research Foundation

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.

Today was Researcher Day, at the 4th annual Global CMT Research Convention. Dr. Carsten Bönnemann set the stage for the day with his keynote talk on intrathecal gene therapy. The day continued with sessions from various clinicians and researchers doing groundbreaking work. Dr. Artie Suckow received the Pathfinder Award for his work developing a clinical candidate with DTx Pharma, which was acquired by Novartis last year. Tomorrow kicks off Patient Day where patients and researchers come together to face the challenges of CMT drug development. Join us live tomorrow at 9 AM ET at cmtconvention.cmtrf.org. We hope to see you all there!

Today kicked off the 4th annual Global CMT Research Convention with the young researchers innovation forum- the next generation of CMT researchers tackling the challenges of finding treatments for CMT. Researchers also gathered to present their blitz presentations of new work in CMT research followed by a poster session. Tomorrow is jam packed as researchers will gather to speak about the latest advancements in CMT research. It’s not too late to register! Visit cmtconvention.cmtrf.org to sign up.

Time is running out—there are only 10 days left to contribute to ENDGAME. Every dollar you donate will be matched by a generous donor, up to $1 million—but only until 12:00 AM on September 27. Turn $100 into $200 Turn $500 into $1,000 Turn $5,000 into $10,000 These next few days are critical. Act now to take advantage of the match and double your impact in the fight against CMT1A. Together, we can close out ENDGAME and celebrate the beginning of clinical trials for CMT1A and, ultimately, the approval of treatments that will end this disease Donate today at cmtrf.org/endgame

We are excited to announce that a generous donor will match every dollar contributed to ENDGAME, up to $1 million, to advance drug development for CMT1A. Your donation will have twice the impact in supporting the critical research needed to end this disease.   Turn $100 Into $200 Turn $500 Into $1,000 Turn $5,000 Into $10,000   We have two weeks left to raise enough funds to close out ENDGAME and call victory on this bold campaign. More importantly, we are excited to celebrate the beginning of the clinical trials for CMT1A and, ultimately, the approval of treatments that will end this disease. Donations must be made between now and 12:00 AM ET on September 27, 2024.   To learn more about ENDGAME, or to donate, visit cmtrf.org/endgame.