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Article

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

Orphanet journal of rare diseases - 19 Mar 2024

Varma Anika, Weinstein Jennifer, Seabury Jamison, Rosero Spencer, Dilek Nuran, Heatwole John, Engebrecht Charlotte, Khosa Shaweta, Chung Kaitlin, Paker Asif, Woo Amy, Brooks Gregory, Beals Chan, Gandhi Rohan, Heatwole Chad

Abstract excerpt

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...

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Identifiers

PMID: 38504253
DOI: 10.1186/s13023-024-03129-6
PMCID: PMC10953228

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

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