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 ...

Owen Donoghue, 44, set out to raise funds for Ella-Rose Kennedy after she was diagnosed with a debilitating progressive brain ...

A Columbia woman is putting a spotlight on a rare, degenerative disease, which targets the nervous system and often impairs ...

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 ...

An Edmond family is on a mission to find a cure. For a decade, Michael Gehr has been battling Friedreich's ataxia, a rare ...

Flynn Mitchell said one the lowest moments of his life was trying to get home safely in his wheelchair in the middle of a ...

Billionaire Graeme Hart's granddaughter Jemima Hawkesby and her friend Flynn Mitchell graduating. She is fundraising to help ...

FA is the most common inherited ataxia, a group of disorders affecting coordination, balance and speech, affecting at least one in every 50,000 people in the UK. Early symptoms of the life-shortening ...

the lack of understanding makes an already devastating diagnosis even more overwhelming for them and their families. Friedreich’s ataxia is a rare, progressive neurodegenerative disease that ...