Friedreich's Ataxia News and Research

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Friedreich's ataxia is an inherited disease that causes progressive damage to the nervous system resulting in symptoms ranging from gait disturbance and speech problems to heart disease. A well-known celebrity afflicted with Friedreich's ataxia is former major leaguer and noted historian Clinton Stewart.

Weill Cornell Medicine researcher wins $2.1 million grant to study repetitive DNA and RNA sequences

Dr. Jeannine Gerhardt, an assistant professor of stem cell biology in obstetrics and gynecology and in reproductive medicine at Weill Cornell Medicine, has received a five-year, $2.1 million grant from the National Institute of General Medical Sciences (NIGMS), part of the National Institutes of Health, for the study of repetitive DNA and RNA sequences and the mechanisms by which they cause cell dysfunction and diseases.

30 Jan 2024

Stem cell transplants may be a promising therapeutic against Alzheimer's, study shows

In the ongoing search for a cure for Alzheimer's disease, a burgeoning branch of medicine is bringing new hope. Stem cell therapies are already being used to treat various cancers and disorders of the blood and immune system.

9 Aug 2023

Transgenic mouse model reveals surprising new connections between peripheral nerves and the heart

Oxidative stress – characterized by elevated levels of unstable molecules called reactive oxygen species– is associated with neurodegeneration and cardiovascular disease.

14 Jun 2023

Using motion capture technology and AI to monitor the progression of movement disorders

A multi-disciplinary team of researchers has developed a way to monitor the progression of movement disorders using motion capture technology and AI.

23 Jan 2023

Study explores cellular alterations in six distinct neurodegenerative diseases

A perplexing range of neurodegenerative diseases are known to attack distinct regions of the brain, causing severe cognitive and motor deficit.

21 Dec 2022

NEJM study shows hidden genetic anomaly behind common late-onset cerebellar ataxia

Researchers at the University of Miami Miller School of Medicine, McGill University and other institutions have found that a well-concealed genetic variation in the gene FGF14, called a DNA tandem repeat expansion, causes a common form of late-onset cerebellar ataxia, a brain disorder that interferes with coordinated movement.

16 Dec 2022

Increasing Awareness Surrounding Ataxia

In this interview, we speak to Dr. Lauren Moore from the National Ataxia Foundation about the condition and the importance of raising awareness for ataxia.

31 Oct 2022

Study addresses multiple challenges of the pathogenesis of Huntington's disease

Recent genetic data from patients with Huntington's disease (HD) show that DNA repair is an important factor that determines how early or late the disease occurs in individuals who carry the expanded CAG repeat in the HTT gene that causes HD.

12 Feb 2021

£1.6 million grant awarded to advance development of MND drug candidate

Researchers from the University of Sheffield's Institute for Translational Neuroscience have been awarded a £1.6 million grant from the Medical Research Council.

26 Jan 2021

Gene therapy used to overcome the cardiac effects of Freidreich’s ataxia

Gene therapy was successfully used to overcome the cardiac effects of Freidreich’s ataxia (FA) in a mouse model of the disease, as reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free online through October 18, 2020.

19 Sep 2020

Developing Vaccines and Therapeutics by Exploiting Structure in Spherical Nucleic Acids

An interview discussing the use of spherical nucleic acids (SNAs) and what they show about the importance of structure in pharmaceutical development.

From Pittcon 2 Jul 2020

Exercise has profound protective effect in mouse models of Friedreich's ataxia

A top exercise researcher at the University of Virginia School of Medicine is urging clinical trials of exercise in patients with Friedreich's ataxia after finding that physical activity has a "profound" protective effect in mouse models of the debilitating genetic disease.

16 Jun 2020

New molecular mechanism can reverse genetic defect responsible for Friedreich's ataxia

Scientists at Tufts University have identified a molecular mechanism that could reverse the genetic defect responsible for Friedreich's ataxia, a neurodegenerative disease that leaves its victims with difficulty walking, a loss of sensation in the arms and legs and impaired speech caused by degeneration of nerve tissue in the spinal cord.

6 Jan 2020

C-Path and FARA announce launch of Friedreich's Ataxia Integrated Clinical Database

Critical Path Institute's Data Collaboration Center and the Friedreich's Ataxia Research Alliance today announced the launch of the Friedreich's Ataxia Integrated Clinical Database.

28 Feb 2019

Newly designed molecule could benefit people with Friedrich's Ataxia

Skin cells taken from patients with a rare genetic disorder are up to ten times more sensitive to damage from ultraviolet A radiation in laboratory tests, than those from a healthy population, according to new research from the University of Bath.

21 Feb 2019

New method to speed up genetic diagnosis of Huntington’s disease

Elongated segments of DNA cause Huntington's disease and certain other disorders of the brain. Researchers funded by the SNSF have developed a method to determine the length of the mutated genes quickly and easily.

10 Jan 2019

Electrical stimulation of the brain may help treat symptoms of neurodegenerative ataxias

Electrical stimulation of the brain and spinal cord may help treat the symptoms of rare movement disorders called neurodegenerative ataxias, according to a study published in the August 22, 2018, online issue of Neurology, the medical journal of the American Academy of Neurology.

23 Aug 2018

Researchers uncover cause of rare childhood neurodegenerative disease

Neurology researchers investigating a rare but devastating neurological regression in infants have discovered the cause: gene mutations that severely disrupt crucial functions in mitochondria, the energy-producing structures within cells.

23 Mar 2018

UCLA researchers reveal reversibility of Friedreich's ataxia in mouse models

In children and adults with Friedreich's ataxia, an inherited disease that causes damage to the nervous system, a loss of coordination typically progresses to muscle weakness and can involve vision, diabetes, and other problems over the course of several years.

20 Dec 2017

Early intervention may be crucial to successful treatment of Friedreich's ataxia

New research published today in Disease Models & Mechanisms indicates that early intervention should be a key target in the development of new therapeutics for Friedreich's ataxia, as current treatments may be administered too late to target the disease effectively.

8 Nov 2017