Simplified classification of inherited metabolic diseases based on pathophysiology: A practical guide for clinicians

Publications
· 5 May 2019
Simplified classification of inherited metabolic diseases based on pathophysiology: A practical guide for clinicians

This paper proposes a simplified classification of inherited metabolic diseases into three main groups—small molecule disorders, complex molecule disorders, and energy metabolism defects—based on clinical and pathophysiological features, to improve understanding, diagnosis, and identification of treatable conditions.