Retrospective assessment of clinical global impression of severity and change in GM1 gangliosidosis: a tool to score natural history data in rare disease cohorts

Retrospective assessment of clinical global impression of severity and change in GM1 gangliosidosis: a tool to score natural history data in rare disease cohorts

Abstract Background Clinical trials for rare diseases pose unique challenges warranting alternative approaches in demonstrating treatment efficacy. Such trials face challenges including small patient populations, variable onset of symptoms and rate of disease progression, and ethical considerations,...

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Main Authors: Connor J. Lewis, Jean M. Johnston, Silvia Zaragoza Domingo, Gilbert Vezina, Precilla D’Souza, William A. Gahl, David A. Adams, Cynthia J. Tifft, Maria T. Acosta
Format: Article
Language:English
Published: BMC 2025-03-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Rare diseases
Clinical trials
GM1 gangliosidosis
Lysosomal storage disorders
Clinical global impressions scale
Online Access:https://doi.org/10.1186/s13023-025-03614-6
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https://doi.org/10.1186/s13023-025-03614-6

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