Poor balance and coordination are prominent features of Friedreich’s ataxia (FRDA). Czech researchers found that neuropsychiatric symptoms are also common among patients with FRDA.

Kronos is cutting 83% of its workforce, while Idorsia is considering cutting as many as 270 jobs. Elsewhere, PTC disclosed ...

BridgeBio’s Attruby wins approval for transthyretin amyloid cardiomyopathy while the FDA accepts Alnylam’s application for ...

New guidance from health technology assessment (HTA) agency NICE backs the use of Wainzua (eplontersen) to treat stage 1 or 2 ...

Don't Miss our Black Friday Offers: Mani Foroohar has given his Buy rating due to a combination of factors including the recent FDA approval of BridgeBio Pharma’s drug Attruby for treating ...

On Nov. 18, the heart failure and transplantation team at Emory University Hospital (EUH) made history, performing the first-ever surgical implantation in the United States of a brand-new type of ...

Imkeldi is a new formulation of imatinib approved as a strawberry-flavored, shelf-stable liquid designed to be more appealing ...