Treatment and trials
Standard care and symptom management
At a glanceThere is no approved treatment that changes the course of CMT2C today, so care focuses on managing symptoms and monitoring breathing, voice and hearing [6]. Good care for CMT2C is team care, with yearly monitoring. No published clinical practice guidelines exist, and recommendations are based on clinical experience [6]. The first drug designed for CMT2C, ABS-0871, has completed a healthy-volunteer study [4], and as of October 2026 its patient trial was listed as planned but not yet recruiting [34].
Your care team
Care involves several specialties and can include neurology, rehabilitation medicine (physiatry), orthopedics, audiology (hearing), laryngology (voice box), pulmonology (lungs), and physical and occupational therapy [6].
The care team guide walks you through each specialist and what to expect at the visit, and includes a checklist you can print.
Managing each problem
| Problem | What standard care offers | Source |
|---|---|---|
| Foot drop and unstable ankles | Shoes with good ankle support, shoe inserts (orthotics), and ankle-foot or knee-ankle-foot braces (orthoses) | [6] |
| Mobility | Forearm crutches, canes, walkers, wheelchairs; exercise within your own limits is encouraged. See Exercise and lifestyle. | [6] |
| Severe high arches or joint contractures (stiff joints that cannot fully straighten or bend) | Orthopedic surgery | [6] |
| Scoliosis | Orthopedic evaluation; spinal fusion is considered if there is pain or the lungs are affected | [6] |
| Tight heel cords | Physical therapy and daily heel cord stretching | [6] |
| Vocal fold weakness (paresis) | Speech therapy; voice box (laryngeal) surgery when needed. Some people benefit from vocal fold lateralization, an operation that moves a vocal fold aside to widen the airway. Some need a tracheostomy, a breathing tube placed through an opening in the neck. In the TRPV4 registry, more than 1 in 10 had needed breathing support from a machine or a tracheostomy [3]. | [6] |
| Breathing problems | Referral to a lung specialist, ideally one who treats neuromuscular disease; night-time ventilation with a bilevel (BiPAP) machine or a home ventilator. CPAP alone does not help weak breathing muscles (see why). See how to spot weak breathing early. | [6] |
| Hearing loss | Rehabilitation options through audiology | [6] |
| Pain and low mood | Treatment of the symptoms | [6] |
| Weight | Nutritional support and weight management | [6] |
Things to avoid
GeneReviews, the main medical reference on TRPV4 disease, advises avoiding four things: obesity, which makes walking more difficult; developing diabetes; medicines that can damage nerves (neurotoxic medications); and upper respiratory tract infections such as colds, which cause vocal fold swelling and worsen airway obstruction [6].
Medication cautions
In July 2023, the Charcot-Marie-Tooth Association (CMTA) replaced its earlier, longer list of medications with “a streamlined list of just two drugs identified as having evidence-backed potential for harm” [35]:
- Vincristine (a chemotherapy drug): potential for severe and sometimes irreversible worsening of CMT symptoms [35].
- Paclitaxel (a chemotherapy drug): associated with nerve damage (neurotoxicity) [35].
The systematic review behind this change found “no convincing evidence” for similar warnings on other drugs, and stressed that people with CMT “should not be denied effective treatments” [36]. The evidence on vincristine comes mainly from people with CMT1A [36]. Medication decisions should be made with a qualified healthcare provider [35].
Pregnancy and family planning
During pregnancy, seeing a high-risk obstetrician or a maternal-fetal medicine specialist is advised [6]. Genetic counseling is recommended to discuss inheritance and its implications [6]. If you are thinking about having children, the Practical tips page sets out every option without steering you toward any of them.