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Development of the natural history component of an early economic model for primary sclerosing cholangitis.
Correlation of retinal vascular characteristics with laboratory and ocular findings in Fabry disease: exploring ocular diagnostic biomarkers.
Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.
🥈 ObservacionalAssociation between one-session bilateral whole-lung lavage and periprocedural complications in patients with pulmonary alveolar proteinosis: a retrospective cohort study.
Economic burden and health related quality of life of ultra-rare Gaucher disease in China.