Short summary of a recent publication, written by scientific experts.
Published: 04 Dec 2018
A novel functional outcome measure for early-onset Charcot-Marie-Tooth disease
- A robust functional outcome measure for Charcot–Marie–Tooth disease (CMT) in infants and children has been developed and validated.
Why this matters
Use of validated clinical trial outcome measures for CMT during infancy and childhood provide an opportunity to treat the disease prior to the onset of long-term disability.