Initial Data from the Ongoing, Blinded Phase 1 Single-Ascending Dose Trial Has Demonstrated Favorable Safety and Pharmacokinetics IND Application Submitted to Expand RESTORE-FA Trial to U.S. Sites; ...

Researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and Friedreich's ataxia. If longer than a certain threshold length, these sequences grow in length ...

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 ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

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

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

The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 for the treatment of Friedreich ataxia (FA ...

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

After a rocky start to the spring, Wall Street came roaring back in May. President Trump’s softened tariff stance reignited ...