New Study Evaluates Peripheral Neuropathy Associated with Chemotherapy in Charcot-Marie-Tooth Disease Gene Variants
source: pixabay.com

New Study Evaluates Peripheral Neuropathy Associated with Chemotherapy in Charcot-Marie-Tooth Disease Gene Variants

Chemotherapy-induced peripheral neuropathy, or CIPN, is a common side effect of chemotherapy. A recent study aimed to uncover whether or not oxaliplatin chemotherapy increased risk of CIPN for those with…

Continue Reading New Study Evaluates Peripheral Neuropathy Associated with Chemotherapy in Charcot-Marie-Tooth Disease Gene Variants
Nonprofit Encourages Charcot-Marie-Tooth Disease Patients to Stay Active
source: pixabay.com

Nonprofit Encourages Charcot-Marie-Tooth Disease Patients to Stay Active

According to a story from Charcot-Marie-Tooth News, a new nonprofit organization called Shark Tooth Cycling has recently begun operations. The mission of the organization is to encourage young people in…

Continue Reading Nonprofit Encourages Charcot-Marie-Tooth Disease Patients to Stay Active
New Study Finds Potential RNA Treatment for Charcot-Marie-Tooth Disease Type 1 A
source: pixabay.com

New Study Finds Potential RNA Treatment for Charcot-Marie-Tooth Disease Type 1 A

CMT1A Charcot-Marie-Tooth disease (CMT) has a few different subtypes. 60% of all patients diagnosed with type 1 of the rare disease have CMT1A. This subtype means patients have an duplicate copy…

Continue Reading New Study Finds Potential RNA Treatment for Charcot-Marie-Tooth Disease Type 1 A

New Marker Found to Track Disease Progression in Charcot-Marie-Tooth Disease Type 1a

A study conducted by researchers from Aix-Marseille University in France has revealed a new way to track disease progression of Charcot-Marie-Tooth disease type 1a (CMT1a)- changes to the nerves within the thigh.…

Continue Reading New Marker Found to Track Disease Progression in Charcot-Marie-Tooth Disease Type 1a
SEPT9 Gene Variant Plays Role in Development of Charcot-Marie-Tooth Disease
source: pixabay.com

SEPT9 Gene Variant Plays Role in Development of Charcot-Marie-Tooth Disease

  According to Charcot-Marie-Tooth News, a recent case study found that a variation of the SEPT9 gene could contribute to the development of Charcot-Marie-Tooth disease. Read the full findings in BMC Medical Genetics. Charcot-Marie-Tooth…

Continue Reading SEPT9 Gene Variant Plays Role in Development of Charcot-Marie-Tooth Disease

This Journal Highlights Current Research on Neuromuscular Diseases (NMD)

In a Special Issue of the journal Disease Models and Mechanisms Highlights, James J. Dowling et al describes pediatric neuromuscular diseases as mostly genetic and affecting areas of the peripheral…

Continue Reading This Journal Highlights Current Research on Neuromuscular Diseases (NMD)
The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others
source: pixabay.com

The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others

The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…

Continue Reading The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others
The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases
source: pixabay.com

The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases

According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…

Continue Reading The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases
An Investigative Therapy for Charcot-Marie-Tooth Disease Type 1A has Shown Lasting Results
source: pixabay.com

An Investigative Therapy for Charcot-Marie-Tooth Disease Type 1A has Shown Lasting Results

Phase 3 Trial A Phase 3 trial run by Pharnext examining the safety and the efficacy of an investigative therapy called PXT3003 began in December of 2015. It enrolled 323…

Continue Reading An Investigative Therapy for Charcot-Marie-Tooth Disease Type 1A has Shown Lasting Results

Experimental Treatment for Facioscapulohumeral Muscular Dystrophy has Disappointing Results but May Still Benefit Charcot-Marie-Tooth Disease

The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…

Continue Reading Experimental Treatment for Facioscapulohumeral Muscular Dystrophy has Disappointing Results but May Still Benefit Charcot-Marie-Tooth Disease

This Company Just Announced Plans to Develop a Gene Therapy for Type 2A Charcot-Marie-Tooth Disease

According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the…

Continue Reading This Company Just Announced Plans to Develop a Gene Therapy for Type 2A Charcot-Marie-Tooth Disease
Study Focuses on Muscle Weakness in Patients with Charcot-Marie-Tooth Disease Type 1A
Pezibear / Pixabay

Study Focuses on Muscle Weakness in Patients with Charcot-Marie-Tooth Disease Type 1A

According to a story from Charcot-Marie-Tooth News, a recent study has revealed that patients with Charcot-Marie-Tooth disease (CMT) type 1A still display signs of muscle weakness even if they retain…

Continue Reading Study Focuses on Muscle Weakness in Patients with Charcot-Marie-Tooth Disease Type 1A
Estela Lugo of the Hereditary Neuropathy Foundation Seeks to Raise Charcot-Marie-Tooth Disease Awareness
harpenz / Pixabay

Estela Lugo of the Hereditary Neuropathy Foundation Seeks to Raise Charcot-Marie-Tooth Disease Awareness

According to a story from Charcot-Marie-Tooth Disease News, Charcot-Marie-Tooth disease is actually somewhat common in the US compared to other rare diseases. In fact, it is estimated that around 150,000…

Continue Reading Estela Lugo of the Hereditary Neuropathy Foundation Seeks to Raise Charcot-Marie-Tooth Disease Awareness
First-of-its-Kind Study of Charcot-Marie-Tooth Disease Type 4J to Begin at UT Southwestern Med Center
Source: Pixabay

First-of-its-Kind Study of Charcot-Marie-Tooth Disease Type 4J to Begin at UT Southwestern Med Center

According to a publication from Charcot-Marie-Tooth News, the first patient has been enrolled in the first ever natural history study of type 4J Charcot-Marie-Tooth disease. Study collaborators — biotechnology company…

Continue Reading First-of-its-Kind Study of Charcot-Marie-Tooth Disease Type 4J to Begin at UT Southwestern Med Center
Study Identifies Differences in Progression and Onset Between Type 4B1 and 4B2 Charcot-Marie-Tooth Disease
Belova59 / Pixabay

Study Identifies Differences in Progression and Onset Between Type 4B1 and 4B2 Charcot-Marie-Tooth Disease

According to a story from Charcot-Marie-Tooth News, a recent study was conducted that compared the presentation of symptoms between two different variants of Charcot-Marie-Tooth disease (CMT): CMT4B1 and CMT4B2. The…

Continue Reading Study Identifies Differences in Progression and Onset Between Type 4B1 and 4B2 Charcot-Marie-Tooth Disease
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
Close Menu