罕见单基因疾病：分子病理生理学和新疗法。
Rare Monogenic Diseases: Molecular Pathophysiology and Novel Therapies.
机构信息
Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy.
出版信息
Int J Mol Sci. 2022 Jun 10;23(12):6525. doi: 10.3390/ijms23126525.
Abstract
A rare disease is defined by its low prevalence in the general population [...].
摘要
罕见病是指在一般人群中的患病率较低的疾病[...]。
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