News

FARA to host April 27 meeting on disease research, management

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…

Gene-editing therapy restores more normal function in FA cell model

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…

Novel compound seen to ease heart muscle disease in FA models

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…

FA likely marked by problems making mitochondrial proteins

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…