Leriglitazone, an oral treatment candidate for Friedreich’s ataxia (FA), is well-tolerated and can significantly prevent ataxia worsening and iron buildup…
Articles by Joana Carvalho, PhD
When present at levels 20 times higher than normal, frataxin became toxic and caused heart problems in a mouse model…
Reata Pharmaceuticals, along with a group of key opinion leaders and the Friedreich’s Ataxia Research Alliance (FARA), have…
Chondrial Therapeutics has merged with Zafgen, giving rise to Larimar Therapeutics, which will continue working to advance …
AVXS-401, an experimental gene therapy for Friedreich’s ataxia (FA), is safe, well-tolerated, and leads to clinically meaningful…
Scientists discovered two microRNAs — tiny RNA molecules that control the activity of several genes — playing a key…
A Phase 2 trial testing leriglitazone as a potential oral treatment for Friedreich’s ataxia (FA) has recruited its…
An individual’s ability to control facial muscles might be better than other tools commonly used to measure disease progression among…
Lab-cultured heart tissues derived from patients’ stem cells are a suitable model to study the progression of Friedreich’s ataxia…
High uptake of iron and copper by cells, caused by an insufficiency of frataxin — the protein lacking in…