| Synonyms ACADVLD, ACYL-CoA DEHYDROGENASE, VERY LONG-CHAIN, DEFICIENCY OF, Acadvl + [38] | 
| Introduction The most severe syndrome in the spectrum of single, large-scale mitochondrial DNA (mtDNA) deletions (SLSMDs), usually presenting shortly after birth with sideroblastic anemia. The condition is often associated with exocrine pancreas insufficiency and multi-system dysfunction including diabetes mellitus, cortisol deficiency, hypothyroidism, hypoparathyroidism, and growth hormone deficiency. Commonly associated clinical findings include the following: failure to thrive, hypotonia, ptosis, ophthalmoparesis, and renal disease. | 
| Target- | 
| Mechanism Stem cell replacements | 
| Active Org. | 
| Originator Org. | 
| Active Indication | 
| Inactive Indication- | 
| Drug Highest PhasePhase 2 | 
| First Approval Ctry. / Loc.- | 
| First Approval Date- | 
| Target | 
| Mechanism PPARδ agonists | 
| Active Org. | 
| Originator Org. | 
| Active Indication | 
| Inactive Indication | 
| Drug Highest PhasePhase 2 | 
| First Approval Ctry. / Loc.- | 
| First Approval Date- | 
| Target | 
| Mechanism ACADVL modulators | 
| Active Org. | 
| Originator Org. | 
| Active Indication | 
| Inactive Indication- | 
| Drug Highest PhasePreclinical | 
| First Approval Ctry. / Loc.- | 
| First Approval Date- | 
| Start Date31 Jul 2023 | 
| Sponsor / Collaborator | 
| Start Date03 Oct 2022 | 
| Sponsor / Collaborator- | 
| Start Date01 Aug 2022 | 
| Sponsor / Collaborator | 
