Use of the experimental gene therapy LX2006 is generally safe and appears to improve or stabilize markers of heart…
Articles by Marisa Wexler, MS
Researchers have developed a new gene therapy for Friedreich’s ataxia (FA) that uses blood cells as delivery vehicles to…
Four weeks of treatment with the highest tested dose of the experimental therapy DT-216P2 was associated with improvements in functional…
Treatment with nomlabofusp, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), leads to increased frataxin protein levels in…
A brain imaging marker linked to increased activity of support cells in the brain may help detect early disease activity…
Friedreich’s ataxia (FA) is characterized by defects in how cells regulate iron, which seem to differ by tissue and…
Following feedback from the U.S. Food and Drug Administration (FDA), Larimar Therapeutics is on track to soon submit an…
Abnormal buildup of iron in mitochondria, the powerhouses of cells, may contribute to heart damage in Friedreich’s ataxia, according…
Scientists at the University of Oxford are embarking on five pioneering research projects — together backed by more than half…
An international team of scientists has modified a standardized neurological assessment so it can better measure the severity of non-movement…