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
Nucleotide metabolism, leukodystrophies, and <scp>CNS</scp> pathologyFrancesco Gavazzi, Laura Adang, Nicholson Modesti et al.|Journal of Inherited Metabolic Disease|2024Cited by 14
Systematic analysis of genotype-phenotype variability in siblings with Aicardi Goutières Syndrome (AGS)Isabella Peixoto de Barcelos, Laura Adang, Sarah Woidill et al.|Molecular Genetics and Metabolism|2024Cited by 12
Early recognition of patients with leukodystrophiesNicholson Modesti, Francesco Gavazzi, Sarah Helen Evans et al.|Current problems in pediatric and adolescent health care|2022Cited by 10
Exploration of Gross Motor Function in Aicardi-Goutières SyndromeFrancesco Gavazzi, Laura Adang, Allan M. Glanzman et al.|Journal of Child Neurology|2023Cited by 9