News

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…

In Charcot-Marie-Tooth disease (CMT), a type of support cell called Schwann cells help to repair nerve injury to limit the severity of the disease, a new study suggests. The finding suggests that therapies aiming to activate certain pathways in these cells may be beneficial in CMT and other peripheral…

Many of the mutated proteins that cause the different forms of Charcot-Marie-Tooth disease type 2 (CMT2) tend to go to a point in nerve cells, or neurons, called stress granules. There, upon stress, they make unwanted interactions with a protein called G3BP, a study found. Breaking these interactions was…

The Charcot-Marie-Tooth Association (CMTA) has given a $225,483 award to support the development of treatments for Charcot-Marie-Tooth disease type 2E (CMT2E). The work is also being supported by the Muscular Dystrophy Association. The project will be led by Mario Saporta, MD, PhD, clinical neurologist and researcher at…

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…

Blocking the immune complement protein C6 sped up the recovery of nerve damage and reduced inflammation in a mouse model of Charcot-Marie-Tooth disease type 1A (CMT1A), but it had no impact on the progressive loss of motor function, a study reports. These data suggest that C6 and related proteins,…

The transmission of signals between the nerves and muscles is impaired in people with Charcot-Marie-Tooth (CMT) type 1 or 2, and the severity of this impairment appears to be related to the disease’s. That’s according to findings from ESTABLISH (NCT04980807), an observational study launched by NMD…

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…