The U.S. Food and Drug Administration (FDA) has put Lexeo Therapeutics’ LX2006, a gene therapy candidate for heart disease in people with Friedreich’s ataxia (FA), on the fast track for development. The award of fast track status aims to expedite the development and regulatory review of investigational therapies…
Patients and caregivers will hear the latest news on Friedreich’s ataxia (FA) research and management at an April 27 meeting in Baton Rouge, Louisiana. The Friedreich’s Ataxia Research Alliance (FARA) will host its FA Research Reception at the Hilton Baton Rouge Capitol Center. Topics for the event…
A gene-editing therapy designed to correct the FXN gene defect that’s associated with Friedreich’s ataxia (FA) reversed several cellular features of the disease in patient-derived nerve cells, according to recent research. Scientists identified that dysfunction of a cellular compartment called the endoplasmic reticulum (ER) might play a key role in…
The use of high-resolution ultrasound in people with Friedreich’s ataxia revealed nerve changes in the armpits and arms that correlated with disease severity in a small study by researchers in Italy. These results suggest that such ultrasounds of the nerves running through the axilla region — the armpits —…
Stealth Biotherapeutics’ investigational therapy SBT-589 showed protective effects against cell energy deficits and heart muscle disease across several preclinical models of Friedreich’s ataxia (FA), including a mouse model of pronounced heart disease, according to new laboratory data. SBT-589 is designed to boost the activity of mitochondria — known…
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…
Get regular updates to your inbox.