A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal ...

A WOMAN with a rare degenerative disease has blasted the Government after patients were left waiting 677 days for access to a ...

An innovative backpack, incorporating aerospace technology, shows promise as a balance aid for patients with the movement disorder ataxia. Research conducted by Radboud university medical center, in ...

Taoiseach Micheál Martin has said he wants to see patients living with Friedreich's Ataxia in Ireland have access to the ...

Intravenous administration of AAVrh.10hFXN, an adeno-associated virus vector encoding normal human frataxin, was associated with reductions in left ventricular mass index (LVMI) and high-sensitivity ...

SOUTH SAN FRANCISCO, Calif., December 15, 2025--(BUSINESS WIRE)--Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own ...

Atop exercise researcher at the University of Virginia School of Medicine is urging clinical trials of exercise in patients with Friedreich's ataxia after finding that physical activity has a ...

To examine the impact of a targeted exome approach for the molecular diagnosis of patients nationwide with a wide range of ataxia-related phenotypes. One hundred and seventy patients with ataxia of ...

AJMC ®: What is your background in treating Friedreich ataxia (FA) in clinical practice and in clinical trials? KUO: I am the director of Columbia University’s ataxia center and have extensive ...

To describe ocular abnormalities in patients with Friedreich ataxia (FRDA). OCT and low contrast test sensitivity show that the visual pathway is affected in FRDA. However, in most patients there is ...

Intravenous gene therapy delivering the frataxin gene appears safe and is associated with preliminary signs of reduced heart damage in patients with Friedreich ataxia cardiomyopathy.