About DT-216P2 DT-216P2 is an improved formulation of a GeneTAC ® small molecule designed to specifically target the GAA repeat expansion mutation that is the underlying cause of FA and restore the ...

Researchers have developed a gene editing strategy that can repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. | Genetics And Genomics ...

Early research shows that editing expanding trinucleotide repeats halts the lengthening process that causes neurological disease.

Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and Friedreich's ataxia.The ...

Autosomal recessive ataxias may present with chorea, but generally onset is in the first two decades. Friedreich ataxia (FA) is by far the most common of these forms, occurring in about 1 in 50 000 ...

There are several current treatment options for Friedreich's ataxia, or FA as I'll call it for the rest of this time. The most important is what we had before the approval of any drugs.

Abstract: Friedreich's ataxia (FA) is an autosomal recessive disorder affecting the nervous and cardiovascular systems, characterized by progressive ataxia, dysarthria, and muscle weakness. It results ...

There are many types, including cerebellar ataxia, sensory ataxia, vestibular ataxia, Friedreich’s ataxia and more general forms of hereditary ataxias. The most common symptoms of the disease ...

Repeat expansion disorders ... These mutations cause a wide range of neurological diseases including spinocerebellar ataxia, Friedreich ataxia, myotonic dystrophy, Huntington’s disease, and ...

Several months after forging a fresh path forward for its troubled Friedreich ataxia (FA) drug vatiquinone, PTC Therapeutics is back with some much-needed positive data. The candidate passed ...