The Muscular Dystrophy Association (MDA) awarded more than $1 million to a new clinical trial (NCT03550300) to evaluate…
Articles by Patricia Inácio, PhD
A proposed therapy for neurodegenerative diseases involving the removal of the neurofilament light chain protein (NEFL) may have harmful consequences…
Researchers found that a neuropathy-based scale may help predict the need for mobility assistance in children with Charcot-Marie-Tooth disease.
A new mouse model that mimics the motor behavior deficits seen in people with Charcot-Marie-Tooth subtype 2O disease (CMT2O) will…
Researchers have identified a new mutation in the MFN2 gene that was linked to early onset of Charcot-Marie-Tooth hereditary neuropathy…
Deficiency in copper, a key factor in several cellular functions, may play a role in Charcot-Marie-Tooth (CMT) disease, say researchers…
Exercising with an anti-gravity treadmill may help patients with Charcot-Marie-Tooth (CMT) disease improve their balance and ability to walk, new…
Falls and near falls are frequent in Charcot-Marie-Tooth disease patients and are the consequence of muscle and joint weakness, according…
Researchers identified a novel mutation responsible for a rare form of Charcot-Marie-Tooth disease in a 13-year-old Chinese patient. The…
Placing the patient and family members at the center of research and drug development in the rare disease field is…