You searched for treatment | Page 2 of 21 | Charcot–Marie

CMTA Leading the Way in CMT1A

CMTA and CMTA-STAR logos on a dark navy background alongside an illustrated roundabout graphic representing the Charcot-Marie-Tooth Association's Strategy To Accelerate Research program.

CMTA is leading a coordinated CMT1A research strategy that connects disease models, drug development, biomarkers, delivery science, and clinical infrastructure to accelerate new treatments.

CMTA research takes the stage at PNS

PNS 2026 showcased CMTA's role in advancing CMT research across the translational pipeline, from biomarkers and natural history studies to clinical trials, therapeutic development, and the scientific collaborations driving progress.

What Filspari Means for People with CMTDIE

FDA approval of Filspari creates a new treatment option for FSGS that may benefit some people living with CMTDIE. Learn why this milestone matters.

CMTA Expands Preclinical Toolbox with New CMT1A Assay

CMTA expands its Preclinical Toolbox with a new CMT1A assay, providing researchers with a human-relevant platform to evaluate potential therapies earlier in drug development.

What 465 People with CMT1A Want Researchers to Know

Results from a patient survey conducted by CMTA, CMT France, and Nervosave Therapeutics highlight what matters most to people living with CMT1A.

Augustine Therapeutics

Augustine Therapeutics’ work builds on the groundbreaking research of Prof. Ludo Van Den Bosch, who...

CMTA Appoints Aimee Yrlas Simpson as Chief Revenue Officer, Expanding Its Impact for People Living with CMT

CMTA appoints Aimee Yrlas Simpson as Chief Revenue Officer, bringing more than 25 years of nonprofit leadership experience to help advance research and support people living with CMT.

Geneticist Behind the First Identified Cause of CMT Joins CMTA-STAR Scientific Advisory Board

Vincent Timmerman, PhD, the Belgian geneticist whose lab identified the first known genetic cause of Charcot-Marie-Tooth disease, joins the CMTA-STAR Scientific Advisory Board. Timmerman brings three decades of CMT genetics research and iPSC disease modeling expertise to CMTA's push to advance therapies into clinical trials.