Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

1 article matching "natural history study"

Clinical trialCLINICALTRIALSMar 26

Trial Now Recruiting: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants (NCT05589714)

Researchers are looking for 1,500 people with inherited eye diseases caused by rare genetic changes to join a study. The study has two parts: first, they'll collect genetic information and eye health data from participants, and second, they'll follow some participants over time to understand how these eye diseases progress. This international study is now accepting new participants.

WHY IT MATTERSIf you have an inherited retinal disease with a rare genetic variant, enrolling in this registry could help researchers understand your condition better and speed up development of future treatments while contributing to a global database of genetic information.
You can act on thisInherited Retinal DegenerationRetinitis PigmentosaRead →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases