Tackling the challenges of product development through a collaborative rare disease network: The foundation fighting blindness consortium

Foundation Fighting Blindness Consortium Investigator Group

Research output: Contribution to journalArticlepeer-review

Abstract

The Foundation Fighting Blindness, a 501(c)(3) nonprofit organization, established an international consortium of inherited retinal disease specialists in 2016, with a mission to accelerate the development of treatments for rare, inherited retinal degenerations, such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, Usher syndrome, choroideremia, and achromatopsia. The Consortium accomplishes its mission by evalu-ating novel outcome measures, sharing standardized study protocols and datasets, and disseminating findings. Having established research infrastructure in the first 3 years, including 39 global research sites, the network is now poised to expand its infrastructure for trials of new therapies in partnership with industry. This model represents an innova-tive approach to overcome challenges of therapeutic development for rare diseases.

Original languageEnglish (US)
Article number23
JournalTranslational Vision Science and Technology
Volume10
Issue number4
DOIs
StatePublished - 2021

Keywords

  • Consortium
  • Genetic
  • Infrastructure
  • Inherited retinal degenerations
  • Natural history
  • Outcome measures
  • Retinitis pigmentosa

ASJC Scopus subject areas

  • Biomedical Engineering
  • Ophthalmology

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