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April 24, 2018April 24, 2018

Targeting Key Protein in CMT Type 2A May Be Basis of Future Therapies, Study Suggests

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Researchers found that small molecules which target the key protein altered in patients with Charcot-Marie-Tooth disease type 2A (CMT2A) may be a viable therapeutic approach and also improve other diseases ... Read more

April 20, 2018April 20, 2018

#AAN2018 – Autonomic Nerve Damage Is Widespread and Greatly Affects CMT Patients, Study Shows

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Damage in the nerves that control involuntary body functions — known as autonomic nerve damage — is highly prevalent and has a significant impact on Charcot-Marie-Tooth disease (CMT) patients, ... Read more

April 17, 2018April 18, 2018

#AAN2018 – Neuropathy-based Scale May Help Predict Mobility Needs in Children with CMT

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Researchers found that a neuropathy-based scale may help predict the need for mobility assistance in children with Charcot-Marie-Tooth disease. The results will be presented April 23 at the 2018 ... Read more

April 16, 2018April 16, 2018

Clinical Trial to Test New Tool for Assessing CMT’s Severity in Children

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The Inherited Neuropathies Consortium will conduct a clinical trial of a tool that researchers developed to assess the severity of children’s Charcot-Marie-Tooth Disease. Scientists say the assessment tool used in adults ... Read more

April 13, 2018April 13, 2018

Pharnext, Developer of CMT1A Treatment Candidate PXT3003, Raises 16 Million Euros

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Pharnext SA — the French biopharmaceutical company developing PXT3003 as a therapeutic candidate to treat Charcot-Marie-Tooth disease type 1A (CMT1A) — announced that it has successfully raised 16 million euros, ... Read more

April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

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A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 11, 2018April 11, 2018

Balance Walking for CMT May Help Patients Stay Fit, Be Healthier

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Like many of us, people living with Charcot-Marie-Tooth (CMT) disease make resolutions to become healthier and more fit. But while starting something new can be exciting, finding out what exercises ... Read more

April 10, 2018April 10, 2018

Protein Component May Lead to More Severe Case of CMT4F, Mouse Study Suggests

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A specific portion of a protein contributes to the stability of myelin, the coating that protects nerve cells, according to a mouse study that has implications for Charcot-Marie-Tooth (CMT) disease. ... Read more

April 6, 2018April 6, 2018

Sox2, Id2 Proteins Play Protective Role in Preventing Cell Damage, CMT1B Mouse Study Finds

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Researchers have found that two proteins, Sox2 and Id2, play a protective role in preventing further damage in specialized nervous system cells of a mouse model of Charcot-Marie-Tooth disease ... Read more

April 4, 2018April 4, 2018

Mouse Study Reveals HERC1 Protein’s Role in Myelin Production in CMT, Other Disorders

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Researchers found that HERC1 protein may be important for the production of myelin, the protective layer in peripheral nerves. This discovery, from a mouse study, increases scientific knowledge about the ... Read more

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