HD Therapeutics - CHDI Fifth Annual Conference

IDrugs. 2010 Apr;13(4):219-23.

Abstract

The CHDI Fifth Annual HD Therapeutics Conference, held in Palm Springs, CA, included topics covering new therapeutic developments in the field of Huntington's disease (HD). This conference report highlights presentations on biomarkers in HD; emerging topics in drug targeting, such as the lysosomal degradation pathway and target prediction by network-based modeling; understanding phenotype and neuronal circuit dysfunction in animal models; regulation of huntingtin protein expression and function; RNAi and antisense technology to deplete the mutant huntingtin protein; and small-molecule drugs that are progressing quickly through the clinic. Investigational drugs discussed include ALN-HTT (Alnylam Pharmaceuticals Inc/Medtronic Inc), EPI-743 (Edison Pharmaceuticals Inc), LNK-754 (Link Medicine Corp) and pridopidine (NeuroSearch A/S).

Publication types

  • Congress

MeSH terms

  • Animals
  • Biomarkers / metabolism
  • Disease Models, Animal
  • Dopamine / metabolism
  • Drug Delivery Systems / methods
  • Enzyme Inhibitors / pharmacology
  • Enzyme Inhibitors / therapeutic use
  • Farnesyltranstransferase / antagonists & inhibitors
  • Humans
  • Huntingtin Protein
  • Huntington Disease / diagnosis
  • Huntington Disease / metabolism
  • Huntington Disease / physiopathology
  • Huntington Disease / therapy*
  • Leigh Disease / drug therapy
  • Leigh Disease / genetics
  • Lysosomes / metabolism
  • Models, Biological
  • Nerve Tissue Proteins / genetics
  • Nerve Tissue Proteins / metabolism
  • Neurodegenerative Diseases / drug therapy
  • Nuclear Proteins / genetics
  • Nuclear Proteins / metabolism
  • Oligonucleotides, Antisense / genetics
  • Oligonucleotides, Antisense / therapeutic use
  • Phosphorylation / drug effects
  • Piperidines / therapeutic use
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / therapeutic use
  • Sheep
  • Sirtuin 1 / genetics
  • Sirtuin 1 / metabolism
  • Ubiquinone / analogs & derivatives
  • Ubiquinone / therapeutic use

Substances

  • Biomarkers
  • Enzyme Inhibitors
  • HTT protein, human
  • Huntingtin Protein
  • Nerve Tissue Proteins
  • Nuclear Proteins
  • Oligonucleotides, Antisense
  • Piperidines
  • RNA, Small Interfering
  • Ubiquinone
  • Farnesyltranstransferase
  • SIRT1 protein, human
  • Sirtuin 1
  • pridopidine
  • Dopamine