Charcot-Marie-Tooth disease

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 ultrasound can accurately diagnose mild to moderate carpal tunnel syndrome (CTS) by measuring the median nerve. Researchers compared ultrasound results with standard nerve tests and checked how they related to pain, symptoms, and quality of life in 75 pe…

This study tested whether a TENS device, which sends mild electrical pulses through the skin, can reduce leg pain in people with Charcot-Marie-Tooth disease. Ten adults with chronic pain participated. The goal was to see if pain scores dropped by at least 30%.

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 surgery to treat Complex Regional Pain Syndrome (CRPS) in the upper limb. 74 adults with CRPS who had not improved with other treatments received either nerve decompression or correction of a specific structural problem. Pain levels were measured before surgery,…

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 tested whether a high-intensity exercise program, run by physical therapy students, can help people with neurologic disabilities move better in everyday life. Fifteen participants with conditions like cerebral palsy, stroke, or spinal cord injury completed the program.…

This study tested three different injection techniques using a sugar-water solution (5% dextrose) to treat mild to moderate carpal tunnel syndrome. 74 adults received one of the methods, and researchers measured pain, tingling, and hand function over three months. The goal was to…

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 completed study looked at how muscle oxygen levels change during exercise in people with various neuromuscular diseases compared to healthy volunteers. Seventeen participants did a knee-extension exercise while a device measured muscle oxygen. The goal was to understand diff…

This study looked at how neck pain changes the way people walk and control their movements. Researchers tested 60 adults—some with chronic neck pain and some without—while they performed walking tasks. The goal was to understand how neck problems affect balance and coordination d…

This completed survey study asked 131 people with or at risk for Huntington disease about their feelings, attitudes, and beliefs toward clinical research. The goal is to help researchers design better studies that are more aware of participants' needs. No treatment or drug was te…

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 whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain diseases that are hard to identify. Researchers enrolled 236 children with white matter abnormalities on brain scans but no known gen…

This study created and tested a new scale called the CMT Pediatric Scale to measure symptoms and physical function in children with Charcot-Marie-Tooth disease. Researchers enrolled 500 children under 21 with CMT types 1, 2, or 4 at multiple centers. The scale assesses foot and h…

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…