The first patient has been dosed in a long-term, open-label extension (OLE) of a Phase 2 dose escalation study that…
Articles by Margarida Maia, PhD
A small molecule called honokiol was seen to restore the function of mitochondria, the small structures producing energy in cells,…
A yet unreported mutation that involves the loss of a large piece of the FXN gene, which is linked to…
Microglia, the brain’s resident immune cells, may go awry in Friedreich’s ataxia (FA) and contribute to lasting inflammation in…
Coenzyme Q10 (CoQ10), an antioxidant that occurs naturally in the body and is found in some foods, may help to…
Three researchers in the U.S. have each been awarded a grant from the Friedreich’s Ataxia Research Alliance (FARA) to…
CTI-1601, an experimental therapy by Larimar Therapeutics, is generally well tolerated by people with Friedreich’s ataxia (FA) and…
People with Friedreich’s ataxia (FA) who also have diabetes, a condition that causes their blood glucose (sugar) level to…
A four-week Phase 2 clinical trial that’ll be testing CTI-1601, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA),…
The Phase 1 clinical program of DT-216, Design Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), remains on track,…