CMT Research Foundation

The greatest gift Maximiliano Barrientos could receive is a cure for CMT. Maximiliano’s CMT diagnostic journey was not an easy one. After multiple medical visits and confusion with other conditions, he finally received a diagnosis after three years. After his diagnosis, Maximiliano became actively involved in finding a cure — something he feels very optimistic about. To him, a cure would mean a better quality of life for millions around the world. Christmas Eve 2021 holds a special place in Maximiliano’s heart as his newborn daughter came home and it was the first Christmas spent with her. Would you please consider commemorating Maximiliano’s favorite holiday year of 2021 by donating $20.21, $202.10 or even $2,021. Your donation will be DOUBLED so that he can receive his greatest gift of all — a cure for CMT. https://lnkd.in/dY8aGe7K

The greatest gift Adam Kauffman could receive is a cure for CMT. CMT has been a part of Adam’s life since childhood. Running through generations of his family, he grew up with the notion that he was defined by CMT. Because of CMT, Adam had to focus his attention on academics. While in the long run, that turned out to be a blessing, it was very difficult to come to terms with as a kid who just wanted to participate in all activities. As he got older, he began questioning why anyone should have to live with it, fueling his drive for a cure. For Adam, a cure would mean freedom for future generations, allowing them to choose their own paths without CMT's limitations. Thanksgiving 2012 was a holiday to remember for Adam as it was the first Thanksgiving after his youngest son was born. Surrounded by family, including his mother and mother-in-law who are no longer with him, he cherishes this memory as a time of togetherness and gratitude. Would you please consider commemorating Adam’s favorite holiday year of 2012 by donating $20.12, $201.20 or even $2,012. Your donation will be DOUBLED so that he can receive his greatest gift of all — a cure for CMT. https://lnkd.in/dY8aGe7K

The greatest gift Luca Haik could receive is a cure for CMT. Living with CMT means that Luca begins his day putting on his leg braces and heading to physical therapy so he can get stronger. CMT makes everyday things difficult — his legs often hurt, and he gets very tired. For Luca, a cure would mean other kids like him could live a life free from the pain of CMT. Christmas 2023 was special for Luca as he was able to spend time with his family and help his mom put up the Christmas tree. He also enjoyed giving presents to his brothers. Would you please consider commemorating Luca’s favorite holiday year of 2023 by donating $20.23, $202.30 or even $2,023. Your donation will be DOUBLED so that he can receive his greatest gift of all — a cure for CMT. https://lnkd.in/dY8aGe7K

The greatest gift Rob Prior could receive is a cure for CMT. For Rob, the hardest part about CMT is watching it slowly take parts of himself and seeing the impact it has on loved ones. In this video, Rob recalls a final precious holiday with his mom, who had bravely battled CMT for most of her life. Wheelchair-bound for years, she passed away from complications shortly after the season ended. For Rob, a cure would mean a life free from the fear and uncertainty that CMT brings, a chance to build a family without the worry of passing this condition on, and a wonderful way to memorialize his mother. Would you please consider commemorating Rob’s most memorable holiday year of 2023 by donating $20.23, $202.30 or even $2,023. Your donation will be DOUBLED so that he can receive his greatest gift of all — a cure for CMT. Learn more: https://lnkd.in/dY8aGe7K https://lnkd.in/gJeAGgYD

The greatest gift that the more than 3 million people worldwide living with Charcot-Marie-Tooth disease could receive this holiday season is a cure. This holiday season, we invite you to explore the deeply personal stories of CMT patients — like Rob Prior, Gary Berg, Jillian Cabernel and Lily Sander — as they share their journeys with CMT, the struggles that they’ve faced and the holiday memories they hold most dear. Each memory has a special year attached to it — one that meant something unforgettable to the storyteller. As you connect with these stories, please consider commemorating the year that resonates with you by making a donation in their honor. Thanks to the Bentzin Family Foundation, all donations will be matched dollar-for-dollar, up to $100,000 through December 31, doubling your impact! Your gift, no matter the size, will go directly towards finding a cure for CMT. Together, we will give CMT patients around the world their greatest gift of all — a cure for CMT. Learn more at https://lnkd.in/gXJ33WGU

CMTRF is pleased to announce that we have invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate therapeutics for CMT. Oligonucleotide therapeutics offer promise for treating genetic disorders but have faced challenges as potential drugs for CMT due to limited delivery to the peripheral nerves and affected cell types. Transferrin receptor (TfR1) antibodies have recently been shown to enhance oligonucleotide delivery to muscle, brain and peripheral nerves. This project by 82VS aims to combine Alloy Therapeutics' novel TfR1 antibodies with oligonucleotides to improve delivery and efficacy in CMT-relevant tissues. If successful, this approach could enable treatments for all types of CMT. Read more here: https://lnkd.in/gbA9tD7p

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 [email protected].

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