According to a story from Charcot-Marie-Tooth news, researchers have found that models of mutations affecting the mitofusin 2 protein (MFN2), which is the protein that is affected Charcot-Marie-Tooth disease type…
Charcot-Marie-Tooth disease (CMT), is a rare neurological condition. It's hereditary, and it affects the way nerves communicate with muscles. There are different types of CMT, determined by which genes are…
According to a story from Digital Journal, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that its investigational drug candidate ACE-083 was granted Fast Track designation from the US Food…
A recent article published by the University of California San Francisco looks back at the development of gene therapy technology and explores how scientists at the university are working to…
All things Levi. Such a generally fun-loving little boy, yet such a complex bundle of medical conflagrations. All blended and tied up in a robust package that makes Levi a…
According to a story from Charcot-Marie-Tooth News, a recent meeting between the US Food and Drug Administration (FDA) and patient advocacy groups took place to discuss the future development of…
Encouraging topline results from a pivotal Phase 3 study into a drug for Charcot-Marie-Tooth type 1A disease have been shared by Pharnext. To find out about this news in more…
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…
According to a story from Charcot-Marie-Tooth News, a recent study has managed to identify new clinical evaluations that will allow doctors to more effectively monitor the condition of patients with…
A recently published study in the Journal of Biosciences and Medicines has concluded that orthoses may help to improve the gait and balance of patients with Charcot-Marie-Tooth disease. Orthoses are…
According to a story from Charcot-Marie-Tooth News, a recent study suggests that changes in the levels of two proteins in the body could serve as biomarkers to indicate the progression…
An ongoing Phase 3 clinical trial program is investigating the drug PXT3003 as an experimental treatment for Charcot-Marie-Tooth disease type 1A in adults. Pharnext SA says that they expect to…
According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…
Researchers have found a new way to monitor disease progression in people with Charcot-Marie-Tooth disease type 1 (CMT1), reports Charcot Marie Tooth News. CMT1 is an inherited genetic condition that…
May 12th through May 16th is National Neuropathy Awareness Week! It is reported that about 1 in 15 Americans are afflicted by neuropathy - but many of them do not…
Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…
Collaboration often leads to greater success. Two advocacy groups, the Hereditary Neuropathy Foundation and True Reply have done just that to benefit patients with Charcot-Marie-Tooth disease. Their combined efforts focus…
A story from Charcot-Marie-Tooth News highlights the necessity for genetic testing and a physical examination prior to chemotherapy in order to prevent nerve damage in people with asymptomatic Charcot-Marie-Tooth disease…
According to a story from news-medical.net, researchers from The Scripps Research Institute have successfully outlined a new path for the treatment of Charcot-Marie-Tooth disease subtype 2D (CMT2D). The scientists hope…
According to a recent study, low copper levels could have a hand in leading to Charcot-Marie-Tooth disease, Charcot-Marie-Tooth News reports. Charcot-Marie-Tooth disease (CMT) is a neurological disorder that affects the…
A new study uncovered two proteins that may be able to reduce symptoms in certain diseases. The proteins may be especially effective at treating diseases with nonsense mutations, such as…
Do you love cheese? Have an irrational fear of cats? Are able to wiggle your nose? As humans, we share more than a few surface-level characteristics with the common house…
At some point in your life, you might have taken a melatonin supplement from a bottle to get to sleep, or to get over jet-lag. Your body naturally produces melatonin…
In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy held an essay contest for tickets to a Charcot-Marie-Tooth disease event. We asked members of the Charcot-Marie Tooth (CMT) community to tell…
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