Administration of the High-Density Lipoprotein Mimetic CER-001 for Inherited Lecithin-Cholesterol Acyltransferase Deficiency

Ann Intern Med. 2021 Jul;174(7):1022-1025. doi: 10.7326/L20-1300. Epub 2021 Mar 2.
No abstract available

Publication types

  • Case Reports
  • Letter

MeSH terms

  • Adult
  • Apolipoprotein A-I / therapeutic use*
  • Female
  • Genes, Recessive
  • Glomerular Filtration Rate
  • Humans
  • Lecithin Cholesterol Acyltransferase Deficiency / complications
  • Lecithin Cholesterol Acyltransferase Deficiency / drug therapy*
  • Lecithin Cholesterol Acyltransferase Deficiency / genetics
  • Mutation, Missense
  • Nephrotic Syndrome / etiology
  • Phospholipids / therapeutic use*
  • Point Mutation
  • Recombinant Proteins / therapeutic use*
  • Treatment Outcome
  • Vision Disorders / etiology

Substances

  • Apolipoprotein A-I
  • CER-001
  • Phospholipids
  • Recombinant Proteins