News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

CMTRF Earns Top Rating From Charity Navigator

Dec 10, 2024 | Press Releases

The CMT Research Foundation has been evaluated for the first time by Charity Navigator, the nation’s largest charity evaluator, and earned a Four-Star Rating, the highest possible rating. Charity Navigator’s impartial, third-party evaluation validates CMTRF’s...

The Greatest Gift: Jillian Cabernel’s Story of Strength, Magic and the Hope for a Cure

Nov 18, 2024 | Life with CMT

The Greatest Gift: Jillian Cabernel, Vancouver, BC, Living With CMT December 2023 was a Christmas to remember for Jillian Cabernel. For the first time in her life, she truly felt the magic of the holidays. Jillian has spent her entire life living with...

Laura MacNeill Named New CEO of CMT Research Foundation

Oct 17, 2024 | Press Releases

The CMT Research Foundation has hired Laura MacNeill as Chief Executive Officer, it was announced today by Foundation Board Chair Peter de Silva. Ms. MacNeill succeeds Cleary Simpson, CEO for the past three years, who becomes Vice Chair of the Board. “Ms. MacNeill has...

CMT Research Foundation Surpasses $10 Million Goal of ENDGAME Capital Campaign

Oct 4, 2024 | Press Releases

The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease announced over the weekend at its annual Global CMT Research Convention that it has surpassed its goal of raising $10 million to fund research...

Actio Biosciences Receives Both Orphan Drug and Rare Pediatric Disease Designations From the FDA For Treatment of CMT2C

Aug 9, 2024 | CMT Research Updates, Drug Development

Actio Biosciences recently announced that they have received both orphan drug designation and rare pediatric disease designation for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C) from the U.S. Food and Drug...

Inaugural Zero-K Run Raises Awareness for CMT and CMTRF

Jul 19, 2024 | Life with CMT

When many people think of the word “race” they imagine a 5k, 10k and even a half-marathon. But, what about a new type of run that directly benefits patients with Charcot-Marie-Tooth disease and covers no distance at all—A Zero-K Run. A Zero-K Run is exactly what it...

The CMT Research Foundation’s 2024 Global CMT Research Convention to Gather Renowned Scientific Experts and Patients to Discuss Status of Treatments and Cures for Charcot-Marie-Tooth Disease

Jul 18, 2024 | Press Releases

The CMT Research Foundation recently announced most of the speakers at its upcoming 2024 Global CMT Research Convention to be held September 26-28, in Cambridge, MA. On Thursday, September 26, CMTRF will host a Young Researcher Innovation Forum for CMT Research....

CMT Research Foundation to Launch Grant Program to Support Young Investigators Working on CMT

Jul 9, 2024 | Press Releases

The CMT Research Foundation announced that at its annual Global CMT Research Convention this September, it will host a Young Researcher Innovation Forum for CMT Research. The program will introduce the Young Researcher Innovation Grant and provide a platform to...

NMD Pharma Receives FDA Clearance to Initiate Clinical Trial of NMD670 for CMT

Jun 18, 2024 | CMT Research Updates, Drug Development

NMD Pharma recently announced that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial of NMD670 in patients living with CMT types 1 and 2. NMD670 is a...

CMT Research Foundation to Benefit from a Portion of Ticket Sales from Alan Jackson’s 2024-2025 LAST CALL: ONE MORE FOR THE ROAD TOUR

May 30, 2024 | Press Releases

The CMT Research Foundation (CMTRF), the only nonprofit organization focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease* (CMT), is pleased to announce support from Alan Jackson's LAST CALL ONE MORE FOR THE ROAD TOUR 2024-2025 dates....