The present invention relates to the medicinal use of small organic compounds, in particular amide and urea derivatives, for the treatment of congenital disorders of glycosylation, and in particular PMM2 congenital disorder of glycosylation.
本发明涉及小型有机化合物,特别是酰胺和
脲衍
生物在治疗先天性糖基化紊乱,尤其是PMM2先天性糖基化紊乱方面的药用用途。