Emerging Drug Therapies for Inherited Retinal Dystrophies

Adv Exp Med Biol. 2019:1185:263-267. doi: 10.1007/978-3-030-27378-1_43.

Abstract

Worldwide, 1 in 2000 people suffer from inherited retinal dystrophies (IRD). Individuals with IRD typically present with progressive vision loss that ultimately results in blindness. Unfortunately, effective treatment options are not widely available due to the genetic and clinical heterogeneity of these diseases. There are multiple gene, cell, and drug-based therapies in various phases of clinical trials for IRD. This mini-review documents current progress made in drug-based clinical trials for treating IRD.

Keywords: Clinical trials; Drug-based therapies; Inherited retinal dystrophies.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic
  • Drug Development*
  • Humans
  • Retinal Dystrophies / drug therapy*