Pfizer and REGENXBIO have established a new collaboration to develop gene therapies specifically designed to treat people…
Articles by Alice Melão
Dimethyl fumarate, an approved therapy for multiple sclerosis (MS), may help people with Friedreich’s ataxia by increasing the amount of…
A patient with Friedreich’s ataxia has been dosed with Minoryx Therapeutics’ investigational therapy leriglitazone in the ongoing Phase…
Minoryx Therapeutics announced it will soon launch a Phase 2 trial in Spain to evaluate its lead therapeutic candidate,…
Iron-induced cell death may contribute to the progression of Friedreich’s ataxia, a study in human and mice cells shows. Inhibition…
Adult patients with Friedreich’s ataxia have generalized abnormalities in the structure and function of both left and right ventricles of…
Treatment with histone deacetylase inhibitors (HDACi) can restore the cellular pathways that become impaired upon loss of functional frataxin…
A naturally produced RNA molecule called FAST-1 can prevent the readout of the FXN gene and, consequently, production of the frataxin…
High blood levels of three specific proteins may represent new biomarkers to monitor active immune cells and ongoing nerve cell…
A noninvasive assessment of the nerve structures in the superficial membrane of the eye, known as the cornea, can help…