Scientists have pinpointed a new genetic mutation in the PMP22 gene as the cause of Charcot-Marie-Tooth disease type 3 (CMT3)…
Articles by Patricia Inácio, PhD
Corrective foot surgery lessened pain and improved foot alignment and quality of life in patients with Charcot-Marie-Tooth (CMT) disease,…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Scientist Timothy Hines, PhD, was awarded a grant of $20,000 to establish cell models to study Charcot-Marie-Tooth (CMT) disease…
The first patient has been enrolled in a pivotal Phase 3 trial testing Pharnext’s investigational therapy PXT3003 for Charcot-Marie-Tooth disease…
An RNA-based treatment normalized the levels of the key PMP22 protein as well as motor function and muscle strength in…
The CMT Research Foundation is collaborating with a scientist to identify fat (lipid) nanoparticles that might work as a vehicle…
An international Phase 3 clinical trial investigating Pharnext‘s investigational therapy PXT3003 for treating Charcot-Marie-Tooth disease type 1A (CMT1A) is…
A new study is recruiting adults with Charcot-Marie-Tooth disease (CMT) type 1A to find better methods…