Is a Treatment for CMT in the Works?

Jill Price avatar

by Jill Price |

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I burned my finger the other day. I didn’t feel it right away, which is not that odd of an occurrence for me.

Often, I don’t feel the heat and don’t realize I have burned myself while cooking. I have Charcot-Marie-Tooth (CMT) type 1A, which may affect the feeling in my extremities.

CMT is also known as hereditary motor sensory neuropathy or peroneal muscular atrophy. Faulty genes may damage nerve cells or interfere with the formation of myelin, an insulating material that protects electrical signals as they travel through the body’s nervous system, including to the arms and legs.

When the myelin sheath is damaged, nerve impulses can slow or stop. That may explain why it can take 30 seconds for me to feel pain after stubbing my toe or burning myself. It would be nice if there was a way to stop that effect of CMT.

There is ongoing research on ways to treat CMT. One recent study claimed that varying amounts of lecithin seemed to ease the course of the disease, but this has not been tested on humans.

Pharnext is sponsoring clinical trials for PXT3003, a combination of baclofen, naltrexone, and sorbitol. According to the manufacturer, PXT3003 has shown positive effects on muscle cells, neuromuscular junctions, and immune cells. In current studies, the investigational medication’s long-term safety is being researched. Meanwhile, earlier this year, the U.S. Food and Drug Administration granted fast track designation for PXT3003’s development.

This is an exciting time for CMT1A patients. The research is promising. In preclinical trials, PXT3003 inhibited the overexpression of the PMP22 gene and eased neuromuscular symptoms. In clinical trials, patients showed improvements. The amount of research and the positive results give us hope that a treatment may be on the horizon.

For now, only mobility aids, physical therapy, and medication can help ease the pain. These help us manage everyday living, but don’t target the cause of the disease.

I am excited to see where the research leads. My hope is that these treatments are found to be effective and can help those of us with CMT1A. I would be happy to have medication available that stops the progression of my symptoms. It would be even better if the effects could be reversed, but I know that is asking a lot.

I would settle for not burning my fingertips when cooking.


Note: Charcot-Marie-Tooth News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Charcot-Marie-Tooth News or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Charcot-Marie-Tooth.


Michelle avatar


Can you advise where to go for testing? Cmt results “inconclusive”but only tested for certain strands. All symptoms mirror those for cmt. Thanks.

sarah Attridge avatar

sarah Attridge

This pxt 3003 treatment is only safe for cmt 1 patients...
some of my family have cmt 2 N it is very rare but cmt is to do with the axaon and it is deeper into the nerve..

please research a treatment for this also..

Michelle avatar


Can you advise where to go for testing? Cmt results “inconclusive”but only tested for certain strands. All symptoms mirror those for cmt. Thanks. (Uk)

Jill Price avatar

Jill Price

My advice is to talk to your doctor, maybe see a neurologist.

Tugba Mendez avatar

Tugba Mendez

Hi Jill, do you have any updated information about PXT3003? I have CMT 1a. I live in Turkey and unfortunately here they say there is no cure since it is genetic... if you have any info and share with me I would be greatful, 🙏


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