Charcot-Marie-Tooth disease type 1

This completed early trial tested a single intravenous dose of EN001 stem cells in 3 adults with Charcot-Marie-Tooth disease type 1E, a severe hereditary nerve disorder with no approved treatment. The main goal was to check safety, including infusion reactions, while also looking…

This early-phase trial tested the safety of a gene therapy called Engensis (VM202) in 12 adults with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder that causes muscle weakness. Participants received multiple injections into their leg muscles and were monito…

This study tested whether a diode laser can act as a biomarker to measure neuropathic pain in people with peripheral neuropathy. Researchers compared pain responses to a lidocaine patch versus a placebo patch in 75 participants. The goal was to see if the laser test could disting…

This study tested whether breathing pure oxygen in a pressurized chamber (hyperbaric oxygen therapy) can help adults with persistent post-concussion symptoms like headaches, fatigue, and memory problems. Twenty participants with mild traumatic brain injury received either real tr…

This study tested a new medicine called NMD670 in 81 adults with Charcot-Marie-Tooth disease types 1 and 2. Participants took either the drug or a placebo twice daily for 21 days. The main goal was to see if the drug could improve walking distance in 6 minutes. The study also che…

This study involved 156 people with Charcot-Marie-Tooth disease type 1A (CMT1A), a rare nerve condition that slowly gets worse. Researchers took blood and skin samples over two years to find biological markers that could show how the disease progresses. The goal was to develop mo…

This study looked at how two types of nerve diseases—inherited and acquired—affect walking and balance. Researchers used motion analysis and simple tests like the Timed Up and Go test in 67 participants. The goal was to find clear differences that could help doctors tell the two …

This study looked at whether measuring a protein called NfL in the blood or spinal fluid can help doctors manage hydrocephalus, a condition where too much fluid puts pressure on the brain. Researchers studied 41 adults with this condition to see if NfL levels relate to symptoms a…

This completed study examined 129 older adults having elective orthopedic surgery under regional anesthesia. Researchers measured cognitive frailty and collected blood and spinal fluid to look for biomarkers of brain inflammation and degeneration. The goal was to see if these bio…