There are currently no approved drugs for the treatment of CHM. Information presented on this page regarding drug research could possibly have application for CHM, or not. Preclinical studies are being undertaken on readthrough drugs for CHM due to nonsense mutation of the CHM/REP1 gene.

Pharmaceutical and neuroprotective therapies ARVO Abstracts May 2013.pdf - Details Download 121 KB



Readthrough drugs for nonsense mediated disease - Approximately one-third of CHM patients:


Antisense Oglionucleotides for nonsense mediated disease:

Splice Switch Switching Oglionucleotides:

RNA Interference:



European Collaboration Developing Cross-Cutting, Vision-Saving Therapies - Cyclic guanosine monophosphate, or cGMP targeting:



Other Drug Platforms: http://www.qltinc.com/development/pipeline.htm


Drug Delivery:


Neuroprotectives:

rhNGF

PIGF
  • Protective effects of placental growth factor on retinal neuronal cell damage - J Neurosci Res. 2013 Nov 30 - Inoue Y, Shimazawa M, Nakamura S, Imamura T, Sugitani S, Tsuruma K, Hara H - Molecular Pharmacology, Department of Biofunctional Evaluation, Gifu Pharmaceutical University, Gifu, Japan: http://www.ncbi.nlm.nih.gov/pubmed/24293397

CNTF

GNDF


Unoprostone Ophthalmic Solution (Development Code UF-021) for the Treatment of Retinitis Pigmentosa:
  • Clinical Trial in Phase III: http://clinicaltrials.gov/ct2/show/NCT01786395
  • Publication - Unoprostone reduces oxidative stress- and light-induced retinal cell death, and phagocytotic dysfunction, by activating BK channels - Kazuhiro Tsuruma, Yuka Tanaka, Masamitsu Shimazawa, Yukihiko Mashima, and Hideaki Hara corresponding author - Molecular Pharmacology, Department of Biofunctional Evaluation, Gifu Pharmaceutical University, Gifu, Japan - Mol Vis. Dec. 30, 2011; 17: 3556–3565: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3250378/

Other:


Other discoveries that may have potential application for CHM research: