Grants and Contracts Details
Description
ABSTRACT
This natural history study of patients with PCDH15 disease-causing variants will accelerate the
development of outcome measures for clinical trials. Sensitive, reliable outcome measures of retinal
degeneration will greatly facilitate development of treatments for retinitis pigmentosa due to PCDH15
disease-causing variants. Together these approaches are expected to have an impact on understanding
PCDH15 related retinal degeneration, developing experimental treatment protocols, and assessing their
effectiveness.
The goals and expected impact of this natural history study are to:
1. Describe the natural history of retinal degeneration in patients with biallelic disease-causing variants in
the PCDH15 gene
2. Contribute to the identification of sensitive structural and functional outcome measures to use for future
multicenter clinical trials in PCDH15 related retinal degeneration
3. Contribute to the identification of populations for future clinical trials of investigative treatments for
PCDH15 related retinal degeneration
Status | Finished |
---|---|
Effective start/end date | 5/5/21 → 6/17/22 |
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