The first patient has been dosed in a long-term, open-label extension (OLE) of a Phase 2 dose escalation study that tested nomlabofusp, formerly CTI-1601, an investigational therapy being developed by Larimar Therapeutics for Friedreich’s ataxia (FA). The OLE study is open to patients who took part in…
Treatment with vatiquinone significantly slowed declines in upright stability among children and young adults with Friedreich’s ataxia (FA) in a clinical trial, leading researchers to predict it could delay the loss of patients’ ability to walk (ambulation) by about nine months. While the MOVE-FA clinical trial failed…
A deficiency of frataxin, the protein whose lack causes Friedreich’s ataxia, leads to problems with mitochondrial translation — the molecular process by which new proteins are made in the mitochondria, the so-called powerhouse of the cell responsible for energy production — according to a new study. The findings specifically…
Voyager Therapeutics has selected a lead gene therapy candidate to advance into clinical trials with the aim of correcting the genetic defect that causes Friedreich’s ataxia (FA). With this decision, which was made with Neurocrine Biosciences, the companies expect to begin clinical trials in 2025. “The nomination…
Use of the investigational therapy CTI-1601, now known as nomlabofusp, was associated with dose-dependent increases in levels of frataxin — the protein whose lack causes Friedreich’s ataxia (FA) — in skin and buccal cells of adult patients in a clinical trial. Treatment tolerability also was reported in top-line…
A small molecule called honokiol was seen to restore the function of mitochondria, the small structures producing energy in cells, and to reduce the number of harmful oxygen-containing molecules in a study that used a cell model of Friedreich’s ataxia (FA). Honokiol, like resveratrol, is a naturally occurring…
The European Commission has approved Skyclarys (omaveloxolone) for people with Friedreich’s ataxia (FA) ages 16 and older, making the treatment the first to win approval for FA in the European Union. The decision comes just a few months after a committee of the European Medicines Agency (EMA) issued…
Gene therapy safely reversed heart problems, lowered blood markers for heart muscle damage, and extended survival in a mouse model of severe Friedreich’s ataxia (FA), a new study shows. The findings further support the development of gene therapy for FA patients with severe heart…
Diabetes, heart rhythm problems, and neurological disability were found to be the clinical factors most likely to influence life expectancy in people with Friedreich’s ataxia (FA) in a recent European registry study. Scientists used the information to generate a model, which also included left ventricle dysfunction, that doctors might…
People in the Friedreich’s ataxia (FA) community await the possibility of gene therapies for their disease, with many saying they would participate in gene therapy trials regardless of the potential for side effects or other consequences. Around half of the survey’s 137 respondents expected that such treatment — delivering…
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