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A screen of molecules known as short-hairpin-loop-RNA (shRNA) revealed that certain features of Friedreich’s ataxia can be relieved through two specific shRNA sequences. In the laboratory of Dr. Robert B. Wilson at University of Pennsylvania School of Medicine, researchers conducted a study that exposed cells isolated from Friedreich’s ataxia patients…

The Muscular Dystrophy Association, the world’s leading nonprofit health agency dedicated to save and improve the lives of people with muscle disease amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases, recently announced it awarded some of the world’s most promising and esteemed scientists research grants amounting to $10 million to fund…

The National Ataxia Foundation, an organization dedicated to improving the lives of those affected by ataxia through support, education and research, is counting down the days to the 16th Annual International Ataxia Awareness Day on September 25, 2015, which will help bring together ataxia organizations from all over the globe in…

Originally established in 2001 by two patient organisations: Association Française contre les Myopathies (AFM) and the European Organisation for Rare Diseases (EURORDIS), EuroBioBank is a unique network of biobanks that stores and distributes quality DNA, cell and tissue samples for scientists conducting research on rare diseases (RDs), including neuromuscular…

Retrope, Inc., a privately held clinical-stage pharmaceutical company leading the advance of a revolutionary new unifying theory of aging and degeneration, has just opened patient enrolment for a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of oral drug RT001. The study aims to determine the drug’s safety, tolerability and pharmacokinetic profile,…

The Minneapolis, Minnesota based National Ataxia Foundation and Ataxia organizations around the world have dedicated September 25, 2015 as the 16th annual International Ataxia Awareness Day (IAAD) help raise awareness about ataxia. Most people have likely never heard of ataxia, which refers to a group of rare and often fatal…

Gene-therapy specialist company, AAVLife, recently acquired a patent from the United States Patent and Trademark Office for an experimental treatment of cardiomyopathy caused by the genetic disorder, Friedreich’s ataxia, which affects the nervous system and causes movement problems. People with this condition develop impaired muscle coordination (ataxia) that worsens over time and commonly…

A collaborative team of international researchers led by the Don Carlo Gnocchi Onlus Foundation, in Milan, Italy have shown that outcomes measured using robotic devices can improve the sensitivity of clinical evaluations for patients with Friedreich’s Ataxia (FA) in regards to dexterity and disease progression over time.  The study, entitled,…

A study published in the journal Nature Medicine reported a promising gene therapy approach for the heart condition associated with Friedreich’s ataxia in an animal model. The study entitled “Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of…

Researchers at the University of Valencia in Spain and Baylor College of Medicine in the United States recently discovered a new pathway involved with Friedreich’s ataxia pathogenesis, providing new therapeutic targets for the disorder. The study was published in the journal PLoS One and is entitled…