Patients with Friedreich’s ataxia (FRDA) who have trouble standing upright are at greater…
Articles by Ana Pena, PhD
Retrotope has dosed the first patient with RT001, an experimental oral treatment for Friedreich’s ataxia (FA),…
Speech disorders like dysarthria are a common and disabling symptom of Friedreich’s ataxia (FA), but they are largely ignored in…
Scoliosis, an abnormal curvature of the spine, is highly prevalent in children with Friedreich’s ataxia (FA), predominantly caused…
Leriglitazone (MIN-102), an investigational oral treatment by Minoryx Therapeutics, has been granted orphan drug status by the…
MOXIe, the Phase 2 study of oral omaveloxolone (omav), is now the first…
MOXIe, the Phase 2 study of oral omaveloxolone that may prove pivotal to it becoming the first approved targeted…
Topline results of Reata‘s MOXIe trial show that almost one year of treatment with the investigational oral…
Friedreich’s ataxia caused by GAA repeat expansion and point mutations — a rare origin of the disease — is…
Treatment with methylprednisolone, an anti-inflammatory medication, had no evident benefit in children and adults with Friedreich’s ataxia (FA) enrolled…