Prospective telemedicine natural history study of multiple sulfatase deficiency
Laura Adang(Children's Hospital of Philadelphia), Francesco Gavazzi(Children's Hospital of Philadelphia), David Isaacs(Vanderbilt University), Lars Schlotawa(University of Göttingen), Rebecca C. Ahrens‐Nicklas(Children's Hospital of Philadelphia), Emily Yu(Children's Hospital of Philadelphia)
Cited by 0
Related Papers
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
|New England Journal of Medicine|2025|272
Janus Kinase Inhibition in the Aicardi–Goutières Syndrome
|New England Journal of Medicine|2020|201
Genetic and phenotypic spectrum associated with IFIH1 gain‐of‐function
|Human Mutation|2020|115
The 2021 European Alliance of Associations for Rheumatology/American College of Rheumatology points to consider for diagnosis and management of autoinflammatory type I interferonopathies: CANDLE/PRAAS, SAVI and AGS
|Annals of the Rheumatic Diseases|2022|97
Comprehensive health assessment for newly arrived refugee children in Australia
|Journal of Paediatrics and Child Health|2004|82