We are presenting 6 cases of Friedreich's ataxia with cardiac changes that were seen within the past few years at the Children's Medical Center of Boston. The autopsied case is the only one of its ...

Friedreich ataxia is an autosomal-recessive, neurodegenerative disease that primarily affects the nervous system and heart. It was named after Nicolaus Friedreich, a Professor of Medicine in ...

Careful monitoring of neurologic function, cardiac function, presence of diabetes, monitoring for scoliosis and visual loss ...

Friedreich's ataxia causes progressive nervous system damage, movement difficulties, and cardiac dysfunction, significantly affecting patient lives. Disclaimer: This is an AI-generated summary of ...

for the treatment of Friedreich’s ataxia (FA). Per the company, SGT-212 is the only full-length frataxin replacement gene therapy candidate that targets the cardiac manifestations of FA.