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Establishing perinatal and neonatal features of Prader-Willi syndrome for efficient diagnosis and outcomes
期刊: EXPERT OPINION ON ORPHAN DRUGS, 2020; 8 (8)
Introduction Prader-Willi syndrome (PWS) is the most common genetic disease causing childhood morbid obesity. Early diagnosis and treatment are utmost......
Epidemiological research, scientific research, and applied research on rare diseases in China: current challenges and prospects for the future
期刊: EXPERT OPINION ON ORPHAN DRUGS, 2017; 5 (1)
Introduction: This review describes the current scientific research on RDs in China. A fundamental flaw in the prevention and control of RDs is the la......