Charcot Marie Tooth Disease News and Research

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Charcot-Marie-Tooth disease is a group of progressive disorders that affect the peripheral nerves. Peripheral nerves connect the brain and spinal cord to muscles as well as sensory cells that detect sensations such as touch, pain, heat, and sound.

Groundbreaking genome-editing technique holds promise for treating Charcot–Marie–Tooth disease

Scientific advances in the last century have changed our world significantly. For example, the world of genetics has opened doors to a myriad of possibilities: augmented human capabilities, cures for diseases, and even changes to the course of evolution.

19 Dec 2023

AI predicts on- and off-target activity of RNA-targeting CRISPRs

Artificial intelligence can predict on- and off-target activity of CRISPR tools that target RNA instead of DNA, according to new research published in Nature Biotechnology.

3 Jul 2023

Researchers provide a framework for unifying and categorizing neurodegenerative diseases based on eight hallmarks

Researchers presented eight hallmarks of neurodegenerative diseases (NDDs), their in vivo biomarkers, and interactions to help categorize NDDs and specify patients within a specific NDD.

21 Feb 2023

New studies point to a broadly applicable treatment for neurodegenerative diseases

Two new studies from Washington University School of Medicine in St. Louis support development of a broadly applicable treatment for neurodegenerative diseases that targets a molecule that serves as the central executioner in the death of axons, the wiring of the nervous system.

26 Oct 2022

Scientists develop RNA-based therapeutic strategy for Charcot-Marie Tooth disease

Charcot-Marie Tooth disease is the most common hereditary neurological disease in the world. It affects the peripheral nerves and causes progressive paralysis of the legs and hands.

9 Mar 2021

Penn researchers identify genetic driver of Charcot-Marie-Tooth disease

Inherited mutations in a gene that keeps nerve cells intact was shown, for the first time, to be a driver of a neuropathy known as Charcot-Marie-Tooth (CMT) disease.

3 Aug 2020

Researchers discover important molecular mechanism of Charcot-Marie-Tooth disease

Charcot-Marie-Tooth (CMT) disease is the most common form of inherited neuropathies. A genetic mutation causes the insulating myelin layer of peripheral nerves to become progressively damaged, resulting in severe disabilities in the case of CMT type 4B, for instance.

8 Jun 2020

Charcot-Marie-Tooth disease study unearths a new mechanism of neurodegeneration

Charcot-Marie-Tooth disease is an inherited neurodegenerative condition that affects 1 in 2500 individuals.

21 Nov 2019

New insight into cause of Charcot-Marie-Tooth disease

The nervous system is the body’s control center, which is extremely fast in decoding messages from the different nerves in the body. This network should be intact and healthy. When something is wrong with the network, it affects the brain’s main functions.

1 Oct 2019

New insights into protein transport could help define strategies for treating eye disease

Many forms of vision loss stem from a common source: impaired communication between the eye and the brain. And at the root of all eye-to-brain communication are the hundreds of proteins generated by the retina's nerve cells.

22 Aug 2019

Schwann cells capable of generating protective myelin over nerves finds research

Researchers have found that Schwann cells, a special type of cells are capable of generating protective myelin sheath covers for the nerves at rates higher than earlier believed.

7 Jul 2019

Scientists unravel how cells unfold proteins

A happy cell is a balanced cell, but for every stupendously twisted protein it creates, it must tear the old ones asunder. That means untangling a convoluted pretzel-like mass for recycling. Cdc48 plays a critical role in unraveling the spent proteins.

29 Jun 2019

Study uncovers potential modifier genes in patients with Charcot-Marie-Tooth disease

Charcot-Marie-Tooth disease is the most common inherited neurological disorder affecting peripheral motor and/or sensory nerves in humans.

18 Jun 2019

UVA researchers make unexpected discovery about irreplaceable nerve cells

Meticulous new research from the University of Virginia School of Medicine has changed a well-accepted scientific belief about neurons, the vital nerve cells that allow us to experience the world and record those experiences as memories in our brains.

22 Nov 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Regenacy Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company developing breakthrough treatments for diabetic and other peripheral neuropathies, today announced a collaboration with the Charcot-Marie-Tooth Association, a registered non-profit organization serving the hereditary neuropathy patient community, to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models.

2 Aug 2018

Newly developed drug compound may help treat Charcot-Marie-Tooth disease

Scientists have developed a new drug compound that shows promise as a future treatment for Charcot-Marie-Tooth disease, an inherited, often painful neurodegenerative condition that affects nerves in the hands, arms, feet and legs.

20 Apr 2018

Families support groundbreaking research on Charcot-Marie-Tooth disease

Lorena Martin's twin daughters kick butt on the soccer field. Martin is proud of how they hold their own, despite having a rare genetic disease called Charcot-Marie-Tooth (CMT), which has damaged their peripheral nervous system. The girls lack proper control of their hands and feet because their nerves can't send signals properly, so they play using power wheelchairs.

9 Apr 2018

TSRI researchers show path to developing treatments for Charcot-Marie-Tooth disease

Scientists at The Scripps Research Institute have now shown a path to developing treatments for disease subtype CMT2D.

8 Mar 2018

HDAC6 inhibition could be promising therapeutic approach for peripheral neuropathies

Inhibiting HDAC6 improves the structural stability of cells and protects against neuronal damage. Leuven research uncovered that targeting this mechanism could be a promising therapeutic approach for peripheral neuropathies, whether due to Charcot-Marie-Tooth disease (CMT) or as a side effect of chemotherapy.

8 Feb 2018