According to a story from PR Newswire, the Hereditary Neuropathy Foundation (HNF) recently announced that it will be conducting a meeting with the US Food and Drug Administration (FDA). This meeting will be a patient focused drug development meeting that will focus specifically on Charcot-Marie-Tooth disease (CMT) and other inherited neuropathies. This meeting is slated for September 28th, in Hyattsville, Maryland.
About Charcot-Marie-Tooth disease
Charcot-Marie-Tooth disease is a hereditary neuropathy which is most characterized by the loss of touch sensation and muscle tissue in certain areas of the body. This disorder is caused by a genetic mutation which typically affects gene PMP22, but sometimes mutations in other genes can also cause a form of the disorder. Some types cause demyelinzation and others affect the neuronal axon. Symptoms include foot drop, muscle wasting and weakness of the legs, hands, and forearms, loss of touch sensation in the arms and legs, foot abnormalities, scoliosis, teeth grinding, squinting, nerve pain, tremors, and problems with swallowing, chewing and speaking. Treatment usually involves maintaining functionality, and may include surgery and physical therapy. Charcot-Marie-Tooth disease does not impact lifespan in the vast majority of cases. To learn more about Charcot-Marie-Tooth disease, click here.
About The Meeting
This meeting will directly involve patients, caregivers, healthcare providers, and industry representatives. It will focus primarily on what challenges many patients with Charcot-Marie-Tooth disease face in order to learn which needs are most urgent and have yet to be met with current therapies. For those who wish to attend but are otherwise unable. A livestream of the meeting will also begin at 8:30am that day.
Allison Moore, who founded and currently leads the HNF, says that the current time is a critical one for the Charcot-Marie-Tooth disease community, as there is a steady pipeline of drugs in the clinical trial stage and ongoing research and development that is expected to continue into 2019. As a result, it is vital that the concerns of patients are heard loud and clear so that effective treatments can be developed.
Additional information and the agenda for this meeting can be found here.
More information about the HNF can be found here.
