News

September 18, 2017

Researchers Identify New Mutation in Gene Linked to CMT Type 4H

News

Researchers identified a novel genetic mutation affecting the FGD4 gene in a patient with Charcot-Marie-Tooth disease type 4H (CMT4H). With an early onset, CMT4 is a slow progressive neurological ... Read more

September 8, 2017

Safety Board OKs Continuation of Phase 3 Trial of PXT3003 as a Charcot-Marie-Tooth Treatment

News

A safety review board has recommended that regulators allow Pharnext to continue conducting its Phase 3 clinical trial of PXT3003 as a treatment for the neurodegenerative disease Charcot-Marie-Tooth type 1A (CMT1A). It was ... Read more

September 6, 2017

Pharnext to Hold Charcot-Marie-Tooth Symposium at Marseille Conference

News

A panel of experts will discuss treatments that are being developed for Charcot-Marie-Tooth Type 1A disease (CMT1A) at a conference in Marseille on Sept. 8. The Charcot-Marie-Tooth symposium, sponsored ... Read more

August 30, 2017August 30, 2017

Flex Pharma’s New CEO William McVicar to Continue Developing CMT Therapy Candidate FLX-787

News

Flex Pharma’s board of directors has named William McVicar, PhD, as the company’s new president and chief executive officer. McVicar will continue supporting the company’s development of new therapies ... Read more

August 25, 2017

CMT-Causing Gene Mutation Found in Study of Chinese Family

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A novel mutation in the PMP22 gene was found in a family with Charcot-Marie-Tooth Disease (CMT) disease, and has been detailed in the study “A Novel Missense Mutation in ... Read more

August 21, 2017August 21, 2017

Researchers in Spain Identify 2 Most Common GDAP1 Gene Mutations that Lead to CMT

News

Researchers in Spain conducted a large study to investigate the incidence of GDAP1 gene mutations leading to Charcot-Marie-Tooth disease (CMT). The study provided useful information on the most common ... Read more

August 18, 2017

Few Surgeons Use the Same Steps to Correct a Foot Deformity in a CMT Patient, Study Indicates

News

A lot of people with Charcot-Marie-Tooth (CMT) disease have surgery to correct foot deformities, a British study reports. Another important finding was that an operation requires a number of ... Read more

August 14, 2017August 14, 2017

Researchers Study Myelin Protein Mutations and Their Links to Charcot-Marie-Tooth Disease

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Researchers have uncovered how mutations in the peripheral myelin protein P2 — recently linked to the development of Charcot-Marie-Tooth (CMT) disease — might impact myelin formation to cause the ... Read more

August 4, 2017

Acceleron Pharma Investigates ACE-083 as Treatment for CMT Patients

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Acceleron Pharma has launched a Phase 2 clinical trial (NCT03124459) to investigate the safety and effectiveness of its product ACE-083 as a potential treatment for Charcot-Marie-Tooth disease (CMT). The ... Read more

August 2, 2017August 2, 2017

French Scientists Report Two Novel Mutations Leading to Inherited Form of CMT

News

French researchers have identified two novel mutations occurring within the NEFH gene that led tof a hereditary form of Charcot-Marie-Tooth disease in two families. Their study, “Cryptic amyloidogenic elements ... Read more