Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD).

ABSTRACT:

Background

Adrenoleukodystrophy (ALD) is a multifaceted, X-linked, neurodegenerative disorder that comprises several clinical phenotypes. ALD affects patients through a variety of physical, emotional, social, and other disease-specific factors that collectively contribute to disease burden. To facilitate clinical care and research, it is important to identify which symptoms are most common and relevant to individuals with any subtype of ALD.

Methods

We conducted semi-structured qualitative interviews and an international cross-sectional study to determine the most prevalent and important symptoms of ALD. Our study included adult participants with a diagnosis of ALD who were recruited from national and international patient registries. Responses were categorized by age, sex,

SUBMITTER: Varma A 

PROVIDER: S-EPMC10953228 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature