Table 3.

Compounds acting on mitochondrial biogenesis and permeability. The table shows the promising experimental results obtained in vitro and/or in vivo after the administration of PGC-1α (to enhance mitochondria biogenesis) and Olesoxime (to regulate mitochondria permeability). The latter one already gave promising results in SMA patients. For clinical trial ID we referred to https://clinicaltrials.gov.

Therapeutic Function	Drug / Molecule	Pathology	Preclinical Studies / PMCID	Preclinical Results	Clinical Trials / Trial ID	Clinical Results
Mitochondrial biogenesis	PGC-1α	PD	Preclinical in vitro and in vivo PMC4293280 [202]	PGC-1α restoration in a cell culture model for α-synuclein oligomerization
				In A30P α-syn transgenic animals: α-synuclein oligomerization reduction
Mitochondrial permeability	Olesoxime	HD	Preclinical in vitro and in vivo PMID: 31283931 [206]	Mitochondrial membrane stabilization; cognitive and behavioral improvement
	Olesoxime	PD	Preclinical in vitro and in vivo PMID: 31283931 [206]	Mitochondrial activity enhancement and apoptosis inhibition
	Olesoxime	SMA	Preclinical in vivo PMC4033913 [204]	Lifespan elongation	Clinical trial Phase 2 Completed PMID: 31283931 [206] NCT01302600	Efficacy in motor improvement and safety have been confirmed
						It could be administered in combinatorial therapy