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A recent article in the neuroscience section of the Medical Express reports the results of a study conducted by researchers in the Charcot-Marie-Tooth/Hereditary Neuropathy Center at internationally acclaimed Cedars-Sinai.…
According to a story from Charcot-Marie-Tooth News, early preliminary testing of a possible gene therapy treatment for a certain type of Charcot-Marie-Tooth disease showed potential in a mouse model. This…
According to a story from fortune.com, the French drug startup Pharnext, led by CEO Daniel Cohen, recently made an announcement not to long ago that made its stock shares rocket…
According to a story from Central Charts, the biopharmaceutical company Acceleron Pharma Inc. recently announced that the company's experimental drug candidate ACE-083 has earned Orphan Drug designation from the US…
According to a story from The Muscular Dystrophy Association (MDA), the organization alongside the Charot-Marie-Tooth Association (CMTA) has awarded grant funding totaling $276,430 to a research study that will develop…
Pharnext SA, a biopharmaceutical company, recently announced results in a PLOS ONE article entitled "Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A…
Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…
According to a story from clinicaltrials.gov, an observational study titled "The Impact of Charcot-Marie-Tooth Disease" was added to the site on December 20th, 2018. The study itself first began on…
"It helps to share experiences with others who understand what I am going through." Jill Price, a mom with Charcot-Marie-Tooth Disease talks about how social media has helped create a…
According to a story from Charcot-Marie-Tooth News, a group of scientists have developed a new scale that can be used to predict disease severity in infants that are diagnosed with…
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…
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