Children with Charcot-Marie-Tooth disease have reduced physical activity and ambulation, factors linked with greater disability, a study suggests. …
Articles by Patricia Inácio, PhD
Two new mutations in the LRSAM1 gene linked to Charcot-Marie-Tooth (CMT) disease type 2P were found in eight…
Further studies are needed to understand how mutations in genes coding for aminoacyl-tRNA synthetases (aaRSs) may help fuel Charcot-Marie-Tooth (CMT)…
ACE-083 has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) to treat …
Independence, mobility, the cost of medical devices and equipment, as well as uncertainty about the future, are the top concerns…
A new mutation in the GARS gene has been associated with autosomal dominant intermediate Charcot-Marie-Tooth disease (DI-CMT), a case report shows.
Two weeks of treatment with Pharnext’s PXT3003 delayed disease onset, corrected motor deficits, and improved dysregulated molecular pathways in a…
Lack of physical activity among children and adolescents with Charcot-Marie-Tooth (CMT) disease has a long-term negative effect on their physical…
Aldevron has signed a long-term agreement with Sarepta Therapeutics to provide the gene-transfer vehicles used in Sarepta’s gene therapies that are…
Charcot-Marie-Tooth disease type X (CMTX) may increase the risk of developing multiple sclerosis (MS), the most common central nervous system…