Pharnext SA — the French biopharmaceutical company developing PXT3003 as a therapeutic candidate to treat Charcot-Marie-Tooth disease type 1A (CMT1A) — announced that it has successfully raised 16 million euros, or $19.7 million, through a private placement. The financial term refers to…
Researchers have found that two proteins, Sox2 and Id2, play a protective role in preventing further damage in specialized nervous system cells of a mouse model of Charcot-Marie-Tooth disease type 1B (CMT1B). The study titled, “Sustained expression of negative regulators of myelination protects Schwann cells from dysmyelination in a…
ACE-083, an investigative therapy for the treatment of Charcot-Marie-Tooth (CMT) disease, was well tolerated and resulted in increased targeted muscle growth in healthy volunteers in a Phase 1 clinical trial, results show. The study, “Locally Acting ACE-083 Increases Muscle Volume in Healthy Volunteers,” was published…
The proteins NME3 and CSNK1G3 can help increase production of proteins lacking in genetic diseases with an underlying nonsense mutation – such as Charcot-Marie-Tooth type 2 disease – leading to a reduction in symptoms, a new study shows. The study “Aminoglycoside-mediated promotion of translation readthrough occurs through a non-stochastic…
Researchers have found that low levels of ceramide, a lipid molecule, leads to changes in mitochondrial structure and function, resulting in the neuronal degeneration seen in CMT2F, a form of Charcot-Marie-Tooth (CMT) disease. The study titled, “Decreased ceramide underlies mitochondrial dysfunction in Charcot-Marie-Tooth 2F,” was published in the…
A lot of people with Charcot-Marie-Tooth (CMT) disease have surgery to correct foot deformities, a British study reports. Another important finding was that an operation requires a number of individual surgical procedures but few surgeons use the same pattern of procedures for the same type of operation. The study, “…
Researchers have identified MCM3AP as a gene involved in the development of a childhood-onset version of the neurological disorder Charcot-Marie-Tooth disease. The study, “MCM3AP in recessive Charcot-Marie-Tooth neuropathy and mild intellectual disability,” was published in the journal Brain. Charcot-Marie-Tooth disease (CMT) is a group of hereditary neuropathies, or diseases affecting…
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