Friedreich's ataxia (FRDA) is a rare, inherited disorder that causes progressive damage to the nervous system. This damage leads to ataxia—problems with movement and coordination—that worsens with ...

Researchers have uncovered a fundamental rule that governs how genes are physically arranged inside the cell nucleus, and how disruptions to that organization can contribute to human disease.

Friedreich's Ataxia is caused by deficiency of frataxin, a mitochondrial protein essential for iron–sulfur cluster biogenesis and cellular energy metabolism. Loss of frataxin disrupts mitochondrial ...

Agilis and Intrexon expect DNA-based therapeutics to provide the ability to target underlying disease mechanisms with precision using tightly-controlled gene therapies for patients with rare inherited ...

Friedreich’s ataxia, or FRDA, is a genetic disorder that progressively damages the nerves and the heart. The affected individuals typically begin to show symptoms between the ages of 5 and 15. They ...

Agilis Biotherapeutics, LLC and Intrexon Corporation have announced an Exclusive Channel Collaboration (ECC) to develop DNA-based therapeutics for Friedreich’s ataxia (FRDA), a rare genetic ...

Friedreich’s ataxia (FRDA) is a triplet repeat expansion disorder with multisystem, progressive symptoms caused by reduced frataxin protein levels and compromised mitochondrial function. Restoration ...