Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approachLaura Ann Adang, Adeline Vanderver, Emily Yu et al.|Molecular Genetics and Metabolism|2024Cited by 25
Determination of Health Concepts in β-Propeller Protein–Associated NeurodegenerationEmma Kotes, Laura Adang, Francesco Gavazzi et al.|Journal of Child Neurology|2024Cited by 6
Clinically Important Endpoints in Individuals With Leukodystrophy: A Multisite StudyEmma Kotes, Adeline Vanderver, Sarah Woidill et al.|Annals of the Child Neurology Society|2025Cited by 0
Functional ability profiles in beta-propeller protein-associated neurodegeneration (BPAN)Francesco Gavazzi, Laura Adang, Samuel R. Pierce et al.|Molecular Genetics and Metabolism|2025Cited by 0