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

José Lopes, PhD avatar

by José Lopes, PhD |

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CMT awareness month

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 by Pharnext, will be part of the 23rd Neuromuscular Days conference, according to a news release. In addition to CMT treatments, it will cover underlying causes of the disease and how it develops.

Pharnext, which is developing a treatment for Charcot-Marie-Tooth, said Dr. David Adams of Université Paris-Sud in Paris will chair the symposium. The speakers will be French neurodenegerative disease experts: Dr. Shahram Attarian of the Assistance Publique Hôpitaux de Marseille; Dr. Yann Péréon of University Hospital and the Reference Center for Rare Neuromuscular Diseases in Nantes; and Dr. Laurent Magy of the Centre Hospitalier Universitaire de Limoges.

CMT is a group of inherited, progressive disorders that cause nerve damage, mostly in they arms and legs. Symptoms typically arise in adolescence or early adulthood.

The CMT1A form of the disease is the most prevalent type of CMT, affecting at least 125,000 people in Europe and the United States. It is caused by an extra copy of the gene PMP22.

A faulty version of the protein that the gene produces leads to the deterioration of myelin, a protective sheath around nerve cells. The degeneration results in nerve damage.

The nerve problems that occur in CMT1A can start as early as the first months of life. The progressive muscle deterioration that is a hallmark of the disease leads to problems walking, running, maintaining balance and using the hands. About 5 percent of patients end up having to use wheelchairs.

At the moment, CMT1A is incurable, and there are no approved medications for its symptoms. Treatment strategies include leg braces, physical and occupational therapy, and surgery.

Pharnext is conducting a Phase 3 clinical trial (NCT02579759) of the safety and effectiveness of PXT3003, a therapy it has developed for CMT1A. Results of previous studies have been promising.

The company designed PXT3003 to prevent over-production of the faulty protein that leads to myelin deterioration, thereby protecting nerve cells.