Patricia Inácio, PhD,  science writer—

Pharnext is analyzing clinical trial data that could support applications to approve PXT3003, its therapy candidate for people with Charcot-Marie-Tooth type 1A (CMT1A), in the U.S. and Europe. “We remain hopeful of finally being able to bring a medical solution to patients with no treatment,” Hugo Brugière, manager…

The country music community is coming together for “A Night at the Opry,” one of many events lined up by the CMT Research Foundation (CMTRF) for CMT Awareness Month this September. The event, held at the Opry House in Nashville at 7 p.m. on Sept. 20, will feature…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Vanda Pharmaceuticals’ VCA-894A for treating Charcot-Marie-Tooth disease type 2S (CMT2S). An orphan drug designation seeks to encourage the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S. — through…

Neuropathic pain due to damage or impairment in the nervous system is common with Charcot-Marie-Tooth disease and linked with heightened social isolation and poorer life satisfaction, according to data from the recent American Academy of Neurology (AAN) annual meeting. The findings suggest “prioritized treatment of neuropathic pain represents a…

A 52-year-old man was diagnosed with Charcot-Marie-Tooth disease type 1 subtype A (CMT1A), after presenting a history of muscle weakness in his legs, foot deformities, and walking impairments. The report illustrates how identifying relatives with similar symptoms may lead to an earlier referral in subsequent CMT cases in the…

The CMT Research Foundation (CMTRF) is joining forces with Augustine Therapeutics to accelerate research into a small molecule therapy for Charcot-Marie-Tooth disease (CMT), with the goal of moving it into clinical testing. With CMTRF and past financial support, Augustine will conduct further studies in its lead, third-generation…

A new report describes the case of a 63-year-old man diagnosed with an atypical, severe form of Charcot-Marie-Tooth disease type 2 (CMT2), with overlapping symptoms of the rarer CMT type 4. Genetic testing showed the man had the subtype CMT2K, caused by mutations in the GDAP1 gene, but with…

Moderate to severe anxiety, depression, and general distress affect up to 25% of people with Charcot-Marie-Tooth disease (CMT), according to data from an Italian national registry. Having a more severe disease correlated with experiencing moderate to severe depression and general distress. Nearly half of patients with mental health conditions…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

The CMT Research Foundation has granted over $500,000 to help develop a gene therapy for Charcot-Marie-Tooth disease type 1B (CMT1B). The grant was awarded to Afrooz Rashnonejad, PhD, principal investigator at the Nationwide Children’s Hospital Center for Gene Therapy and a professor at the Ohio State University’s department of…