Researchers Identify New Mutations Linked to Charcot-Marie-Tooth Disease
skeeze / Pixabay

Researchers Identify New Mutations Linked to Charcot-Marie-Tooth Disease

According to a story from Charcot-Marie-Tooth News, a team of scientists have successfully identified four new mutations that are capable of causing Charcot-Marie-Tooth disease. These mutations affect the GJB1 gene. These new…

Continue Reading Researchers Identify New Mutations Linked to Charcot-Marie-Tooth Disease

The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…

Continue Reading The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

New Research With the Potential to Alleviate Symptoms of Parkinson’s, Alzheimer’s and Charcot-Marie-Tooth Disorders

  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.…

Continue Reading New Research With the Potential to Alleviate Symptoms of Parkinson’s, Alzheimer’s and Charcot-Marie-Tooth Disorders
Gene Therapy Displays Potential for Treating Charcot-Marie-Tooth Disease 4 Subtype C
Kapa65 / Pixabay

Gene Therapy Displays Potential for Treating Charcot-Marie-Tooth Disease 4 Subtype C

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…

Continue Reading Gene Therapy Displays Potential for Treating Charcot-Marie-Tooth Disease 4 Subtype C
Investigational Drug for Charcot-Marie-Tooth Disease Earns Orphan Drug Designation
NeuPaddy / Pixabay

Investigational Drug for Charcot-Marie-Tooth Disease Earns Orphan Drug Designation

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…

Continue Reading Investigational Drug for Charcot-Marie-Tooth Disease Earns Orphan Drug Designation

Study Testing Gene Therapy for Charcot-Marie-Tooth Disease Earns Funding from MDA and CMTA

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…

Continue Reading Study Testing Gene Therapy for Charcot-Marie-Tooth Disease Earns Funding from MDA and CMTA
Pharnext Announces Preclinical Trial Results Affecting Onset of Charcot-Marie-Tooth Disease 1A
source: pixabay.com

Pharnext Announces Preclinical Trial Results Affecting Onset of Charcot-Marie-Tooth Disease 1A

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…

Continue Reading Pharnext Announces Preclinical Trial Results Affecting Onset of Charcot-Marie-Tooth Disease 1A
New Measure Developed to Assess the Severity of Charcot-Marie-Tooth Disease in Babies
1041483 / Pixabay

New Measure Developed to Assess the Severity of Charcot-Marie-Tooth Disease in Babies

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…

Continue Reading New Measure Developed to Assess the Severity of Charcot-Marie-Tooth Disease in Babies
Scientists May Have Found a New Way to Predict The Severity of Type 2A Charcot-Marie-Tooth Disease
DarkoStojanovic / Pixabay

Scientists May Have Found a New Way to Predict The Severity of Type 2A Charcot-Marie-Tooth Disease

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…

Continue Reading Scientists May Have Found a New Way to Predict The Severity of Type 2A Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth: Misconceptions from People Who Just Don’t Know
Source: Pixabay.com

Charcot-Marie-Tooth: Misconceptions from People Who Just Don’t Know

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…

Continue Reading Charcot-Marie-Tooth: Misconceptions from People Who Just Don’t Know
Experimental Treatment for Charcot-Marie-Tooth Disease Gets FDA Fast Track Designation
annca / Pixabay

Experimental Treatment for Charcot-Marie-Tooth Disease Gets FDA Fast Track Designation

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…

Continue Reading Experimental Treatment for Charcot-Marie-Tooth Disease Gets FDA Fast Track Designation

Patient Groups and the FDA Meet For Charcot-Marie-Tooth Disease Drug Development

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…

Continue Reading Patient Groups and the FDA Meet For Charcot-Marie-Tooth Disease Drug Development
A Phase 3 Study Shows Encouraging Results for a Potential Treatment for Charcot-Marie-Tooth Type 1A
DarkoStojanovic / Pixabay

A Phase 3 Study Shows Encouraging Results for a Potential Treatment for Charcot-Marie-Tooth Type 1A

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…

Continue Reading A Phase 3 Study Shows Encouraging Results for a Potential Treatment for Charcot-Marie-Tooth Type 1A
HNF to Meet With The FDA to Discuss Treatments for Charcot-Marie-Tooth Disease
Free-Photos / Pixabay

HNF to Meet With The FDA to Discuss Treatments for Charcot-Marie-Tooth Disease

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…

Continue Reading HNF to Meet With The FDA to Discuss Treatments for Charcot-Marie-Tooth Disease
Orthoses Could Help to Improve Gait and Balance for Patients with Charcot-Marie-Tooth Disease, According to a Recent Study
ashokorg0 / Pixabay

Orthoses Could Help to Improve Gait and Balance for Patients with Charcot-Marie-Tooth Disease, According to a Recent Study

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…

Continue Reading Orthoses Could Help to Improve Gait and Balance for Patients with Charcot-Marie-Tooth Disease, According to a Recent Study
October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease
mohamed_hassan / Pixabay

October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease

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…

Continue Reading October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease
What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?
Source: Pixabay

What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?

Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…

Continue Reading What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?