Charcot-Marie-Tooth disease type 1A
MONDO:0007309Charcot-Marie-Tooth disease type 1A (CMT1A) is a type ofinherited neurological disorder that affects the peripheral nerves. Affected individuals experience weakness and wasting (atrophy) of the muscles of the lower legs beginning in adolescence; later they experience hand weakness and sensory loss. CMT1A is caused byhaving an extra copy (a duplication) of the PMP22 gene. It is inherited in an autosomal dominant manner. Treatment for this condition may include physical therapy ; occupational therapy ; braces and other orthopedic devices; orthopedic surgery;and pain medications.
Also known as: CMT1A, Charcot-Marie-Tooth disease type 1A, Charcot-Marie-Tooth disease, type 1A, Charcot-Marie-Tooth syndrome type 1A, HMSN1A, hereditary motor and sensory neuropathy 1A, microduplication 17p12, CMT 1A
23 clinical trials for this condition and its sub-types, 16 tagged with Charcot-Marie-Tooth disease type 1A itself.
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Gene therapy injection tested for rare nerve disease
Disease control CompletedThis 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…
Phase 1/2 • Sponsor: Helixmith Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Laser test could objectively measure nerve pain for first time
Diagnosis CompletedThis 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…
Sponsor: University of Utah • Aim: Diagnosis
Last updated Jun 27, 2026 12:09 UTC
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Ultrasound probes nerve stiffness in rare nerve diseases
Knowledge-focused CompletedResearchers at Nantes University Hospital are studying whether nerves feel stiffer in people with three rare peripheral neuropathies: CIDP, CMT1a, and anti-MAG neuropathy. They use ultrasound shear wave elastography to measure nerve stiffness in 36 participants aged 18 to 65 and …
Sponsor: Nantes University Hospital • Aim: Knowledge-focused
Last updated Sep 16, 2026 00:00 UTC
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Scientists hunt for clues in blood and skin to speed up CMT treatments
Knowledge-focused CompletedThis 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…
Sponsor: University Medical Center Goettingen • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:29 UTC
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Walking study reveals balance differences in nerve diseases
Knowledge-focused CompletedThis 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 …
Sponsor: University Hospital, Lille • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:03 UTC