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Six-minute walk distance predicting the risk of mortality in lymphangioleiomyomatosis patients.
Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983-2022.
Etiology and outcomes of fetal renal abnormalities in Southern China: a single-tertiary-center study.
Newborn screening facilitates early theranostics and improved spinal muscular atrophy outcome: five-year real-world evidence from Taiwan.
Analysis of SMN protein in umbilical cord blood and postnatal peripheral blood of neonates with SMA: a rationale for prompt treatment initiation to prevent SMA development.