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.

New measurement tool for clinical trials to help children with Charcot-Marie-Tooth disease

An international study led by the University of Sydney and published in the Annals of Neurology has the potential to improve the design of clinical trials for the treatment of Charcot-Marie-Tooth disease, a disorder which affects the peripheral nervous system.

8 May 2012

U-M’s second human embryonic stem cell line now available for federally-funded research

The University of Michigan's second human embryonic stem cell line has just been placed on the U.S. National Institutes of Health's registry, making the cells available for federally-funded research. It is the second of the stem cell lines derived at U-M to be placed on the registry.

26 Apr 2012

Genetically modified mouse mimics key features of Charcot-Marie-Tooth disease

A team of scientists from the University of Missouri created a genetically modified mouse that mimics key features of Charcot-Marie-Tooth disease, an inherited neuromuscular disease affecting approximately 150,000 people in the United States.

18 Apr 2012

U-M's first human embryonic stem cell line to be placed on the U.S.NIH registry

The University of Michigan's first human embryonic stem cell line will be placed on the U.S. National Institutes of Health's registry, making the cells available for federally-funded research. It is the first of the stem cell lines derived at the University of Michigan to be placed on the registry.

15 Feb 2012

Damaged mitochondria equivalent to an environmental disaster in the cell

Current thinking about Parkinson's disease is that it's a disorder of mitochondria, the energy-producing organelles inside cells, causing neurons in the brain's substantia nigra to die or become impaired.

11 Nov 2011

Pharnext puts together €2.5M to fund development of Pleodrug for Charcot-Marie-Tooth disease

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments based on Pleotherapy for severe neurological diseases, today announced that it has put together a €2.5 million round of financing (corresponding to a pre-money company valuation of €60 million).

8 Sep 2011

World's best genetic map now built in African-Americans

A group of researchers from the University of Oxford, Harvard Medical School and the University of Mississippi Medical Center has constructed the world's most detailed genetic map, a tool scientists can use to better understand the roots of disease and how DNA is passed generationally to create diversity in the human species.

21 Jul 2011

Pharnext receives French approval for Pleodrug Phase II clinical trial in Charcot-Marie Tooth disease type 1A

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments for severe neurological diseases and other major unmet medical needs, today announced that the French Agency for Healthcare Product Safety (AFSSAPS) has authorized a Phase II clinical trial with the first drug (a Pleodrug™) generated by the company's Pleotherapy™ technology platform in patients suffering from Charcot-Marie Tooth disease type 1A (CMT1A.

4 Dec 2010

OSEO awards €10.4 million to DIPPAL project targeting Alzheimer's disease

The biotech companies PHARNEXT and BIOSYSTEMS INTERNATIONAL, the Bordeaux Hospital where CMRR is located, the university Bordeaux Segalen and Inserm (the center U897 "Epidemiology and Biostatistics") have joined forces to carry out the DIPPAL2i project. This innovative research and industrial development project is targeting Alzheimer's disease (AD) and has attracted significant financial support from OSEO (the French state innovation agency).

18 Nov 2010

Study finds genetic change in chromosome 4 causes FSHD

The Muscular Dystrophy Association today heralds a landmark muscular dystrophy advance by an international study team of scientists and physicians from the Netherlands, United States, France and Spain. Led by MDA-grantee Silvère van der Maarel, Ph.D., at Leiden University Medical Center in the Netherlands, the collaborative study of more than 2,300 people found that two distinct genetic changes on chromosome 4 must be present to cause facioscapulohumeral muscular dystrophy (FSHD).

21 Aug 2010

Muscular Dystrophy Association awards $14.1M in new grants

The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

20 Aug 2010

MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

18 Aug 2010

Pharnext closes €4.8-million Series A funding round

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments based on Pleotherapy™ for severe neurological diseases, today announced that it had closed a €4.8-million Series A round of funding on June 3rd (corresponding to a company valuation of €45 million).

9 Jun 2010

MDA awards over $21M in grants for advancing critical neuromuscular research

Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multi-year awards to be dispersed over the next three years.

15 Apr 2010

MDA, FFSHR award $200,000 grant for developing FSH dystrophy therapy

The Muscular Dystrophy Association (MDA), headquartered in Tucson, Ariz., and Friends of FSH Research (FFSHR), based in Kirkland, Wash., has jointly awarded a two-year, $200,000 grant to Dr. Joel Chamberlain, a research assistant professor of medical genetics at the University of Washington. The grant, equally funded by the two organizations, will enable the laboratory led by Dr. Chamberlain to study RNA interference as an investigative and therapeutic tool for facioscapulohumeral muscular dystrophy.

7 Apr 2010

MDA awards 42 medical researchers and labs over $21M for critical neuromuscular research

Forty-two medical researchers and their labs have been awarded over $21 million by the Muscular Dystrophy Association to continue critical neuromuscular research. Many of the grants are multi-year awards to be dispersed over the next three years.

1 Apr 2010

Muscular dystrophy: $1M grant for study of erectile dysfunction drugs in improving muscle blood flow

A Cedars-Sinai Heart Institute cardiologist has been awarded a three-year, nearly $1 million grant from the Muscular Dystrophy Association (MDA) to study whether drugs used to treat erectile dysfunction could also be used to improve muscle blood flow and reduce fatigue in muscular dystrophy patients.

26 Mar 2010

Cardiologist receives grant to study impact of erectile dysfunction drugs on muscular dystrophy patients

25 Mar 2010

Scientists identify specific causative genetic mutation associated with CMT

Scientists using advanced genomic analysis technologies from Life Technologies Corporation have sequenced an individual’s genome and identified the specific causative mutation associated with Charcot-Marie-Tooth Disease (CMT), one of the most common inherited neurological disorders currently affecting 1 in 2,500 individuals in the United States.

11 Mar 2010

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.

20 Jan 2010