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.

Update on NMD Pharma Clinical Trial

Nov 18, 2024 | CMT Research Updates

NMD Pharma, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, has announced that it has dosed the first Charcot-Marie-Tooth disease patient in its Phase 2a clinical trial of NMD670...

CMT Research Foundation Funds Project with Alloy Therapeutics’ 82VS to Test Novel RNA Therapeutics for Charcot-Marie-Tooth Disease

Nov 13, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation has 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 (AOC) therapeutics for CMT. Oligonucleotide therapeutics offer promise for...

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...

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...

Augustine Therapeutics Raises $18.5 Million in a Series A First Closing

Jun 26, 2024 | CMT Research Updates, CMTRF Funded Research

The Charcot-Marie-Tooth Research Foundation participated in the $18.5 million Series A funding round recently announced by Augustine Therapeutics. The financing was led by Asabys Partners, with the participation of Eli Lilly & Company, CMTRF, and current investors...

CMT Research Foundation Invests in ReviR Therapeutics Research to Pioneer Small Molecule Therapeutics for CMT1A

Jun 19, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation has formed a strategic partnership with ReviR Therapeutics to advance small molecule therapeutics that modulate the expression of the gene that causes CMT1A. ReviR Therapeutics is developing a class of drugs called small molecule splice...

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...