Fundació Institut de Recerca Biomédica de Bellvitge (IDIBELL);Centro de Investigación Biomédica en Red (CIBER)
发明人:
申请号：
EP16382602.7
公开号：
EP3335730A1
申请日:
2016.12.15
申请国别(地区):
EP
年份:
2018
代理人:
摘要：
The invention relates to a compound of formula (I)as described in the invention, or a pharmaceutically acceptable salt, stereoisomer or solvate thereof, or a mixture thereof for use in the prevention and/or treatment of a disease caused by loss of function of ABCD1 gene, a disease caused by accumulation of very long fatty acids in organs and plasma or of a peroxisomal disorder. The invention also relates to combinations comprising (i) a compound of formula (I) and ii) an antioxidant, an activator of mitochondria biogenesis, an antiinflammatory compound, an activator of autophagy, a peroxisomal biogenesis activator or a mixture thereof, and the medical use of said combinations.
