A single dose of a gene therapy delivering a working copy of GJB1 — the gene mutated in X-linked…
Articles by Marta Figueiredo, PhD
A higher number of daily steps, measured through wearable sensors, was associated significantly with more lower-body strength and functional abilities…
The Hereditary Neuropathy Foundation is supporting preclinical work focused on mutations in the CNTNAP1 gene, which were linked previously to symptoms…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
To promote physical activity in people with Charcot-Marie-Tooth (CMT) disease, the charity CMT UK shared seven exercise videos…
The CMT Research Foundation (CMTRF) wants to turn September from Awareness Month into “CMT Action Month.” This year, the…
Mutations in nine genes associated with Charcot-Marie-Tooth disease type 2 (CMT2) generally lead to similar deficits in muscle structure…
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to PXT3003 for the treatment of…
Acceleron Pharma is discontinuing the clinical development of ACE-083, its experimental treatment candidate for Charcot-Marie-Tooth (CMT) disease,…