Improving quality of life in rare diseases using disease-specific, multidisciplinary online interventions on the example of rare X-linked adrenoleukodystrophy: a randomized-controlled trial.

ABSTRACT:

Background

People with rare diseases (RDs) often require intensive multidisciplinary care in disease-specific centers of excellence (CoE). However, access is limited for most patients living remotely. X-linked adrenoleukodystrophy (X-ALD) is a genetic RD leading to demyelination of the central and peripheral nervous system.

Objectives

This randomized-controlled trial tested the feasibility, acceptance, and effectiveness of a multidisciplinary online intervention provided by a CoE on the quality of life (QoL) and well-being of symptomatic women with X-ALD.

Design

Single-center, randomized-controlled clinical trial involving 68 German-speaking women with symptomatic X-ALD.

Methods

Participants were randomized into an experimental group (EG, n = 34) receivi

SUBMITTER: Schafer L 

PROVIDER: S-EPMC12477361 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature