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Patient-reported, psychosocial and health economic outcomes in mild to moderate Friedreich's ataxia: baseline results of the PROFA studyResearch in context

Summary: Background: Friedreich ataxia (FA) is the most common autosomal recessive ataxia. Little attention has been paid to FA's impact on patient-reported, psychosocial, and health-economic outcomes. This study aimed to report these outcomes across FA's disability stages 1–5. Methods: We assessed...

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Main Authors: Marcus Grobe-Einsler, Stéphanie Borel, Maresa Buchholz, Sabrina Sayah, Rania Hilab, Lucie Pierron, Audrey Iskandar, Brittany Humphries, Claire Ewenczyk, Anna Heinzmann, Mariana Atencio, Katrin Feldmann, Vivian Maas, Jennifer Faber, Sylvia Boesch, Elisabetta Indelicato, Kathrin Reetz, Jörg B. Schulz, Almut T. Bischoff, Thomas Klopstock, Ludger Schöls, Martina Minnerop, Dagmar Timmann, Elin H. Davies, Thomas Klockgether, Alexandra Durr, Feng Xie, Bernhard Michalowsky
Format: Artigo
Language:Inglês
Published: Elsevier 2026-02-01
Series:The Lancet Regional Health. Europe
Subjects:
PROFA
Friedreich
Ataxia
FA
Health
Economic
Online Access:http://www.sciencedirect.com/science/article/pii/S2666776225003448
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