Friedreich’s ataxia (FA) patients with less common types of disease-causing mutations in the FXN gene may have a distinct clinical presentation from patients with a more standard genetic profile, research suggests. A certain group of these mutations associated with higher production of frataxin — the protein lacking in FA — were…
An experimental compound called ATH434, currently being evaluated in clinical trials for a neurological disorder called multiple system atrophy, also may have the potential to treat Friedreich’s ataxia (FA). Data specifically indicate that ATH434 could help to reduce the toxic buildup of iron in cells that is characteristic…
May will be a busy month for patients, doctors, researchers, and advocates in the Friedreich’s ataxia (FA) community as they participate in FA Awareness Month, an initiative for sharing information, personal stories, and support for those living with the rare neuromuscular disease. Named after Nikolaus Friedreich, the German physician…
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…
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