Orphan drugs in development for long-chain fatty acid oxidation disorders: challenges and progress

Article, Review English OPEN
Merritt II, J Lawrence; Sun,Angela;
(2015)
  • Publisher: Dove Press
  • Journal: Orphan Drugs: Research and Reviews (issn: 2230-6161)
  • Related identifiers: doi: 10.2147/ODRR.S63061
  • Subject: treatment | Research and Reviews [Orphan Drugs] | bezafibrate | RM1-950 | Fatty acid oxidation disorders | Therapeutics. Pharmacology | 3-hydroxybutyrate | gene therapy | triheptanoin

Angela Sun, J Lawrence Merritt II Department of Pediatrics, University of Washington, Seattle, WA, USA Abstract: Fatty acid oxidation disorders are inborn errors of metabolism resulting in failure of ß-oxidation within or transport of fatty acids into the mit... View more
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