Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approachLaura Ann Adang, Adeline Vanderver, Anjana Sevagamoorthy et al.|Molecular Genetics and Metabolism|2024Cited by 25
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophyLaura Ann Adang, Maria L. Escolar, Emily Yu et al.|Molecular Genetics and Metabolism|2024Cited by 8
Validation of GMFC-MLD scale as a measure of gross motor function in metachromatic leukodystrophySylvia Mutua, Laura Adang, Anjana Sevagamoorthy et al.|Molecular Genetics and Metabolism|2024Cited by 0
Prospective telemedicine natural history study of multiple sulfatase deficiencyLaura Adang, Francesco Gavazzi, David Isaacs et al.|Molecular Genetics and Metabolism|2023Cited by 0
Development of a rigorous approach for retrospective natural history studies in leukodystrophiesAnjana Sevagamoorthy, Laura Adang, Francesco Gavazzi et al.|Molecular Genetics and Metabolism|2024Cited by 0