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BBS1-related ciliopathy

MONDO:1040043

Any ciliopathy caused by variants in the BBS1 gene.

Also known as: BBS1-related ciliopathy

1 clinical trial for this condition and its sub-types, 0 tagged with BBS1-related ciliopathy itself.

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Where it sits in the disease tree

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Part of

↑ Ciliopathy (49)

Sub-types of BBS1-related ciliopathy

Bardet-Biedl syndrome 1 1 trial

Including sub-types (1) Tagged with BBS1-related ciliopathy (0)

Trials to join now! 1

Gene therapy injection aims to save sight in rare genetic disease

Disease control Recruiting now

This early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to check safety and find the right dose, whi…

Early phase 1 • Sponsor: Axovia Therapeutics • Aim: Disease control

Last updated Aug 06, 2026 00:00 UTC