Bardet-Biedl syndrome

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At a Glance

Live from database

Regulatory Watchboard

4 events
May 2026First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration

Axovia Therapeutics — EARLY_PHASE1

TrialNOT YET RECRUITING
Dec 2025Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome

Federal Research Institute of Pediatric Hematology, Oncology and Immunology — PHASE2

TrialNOT YET RECRUITING
Jan 2024Early Genetic Identification of Obesity

Rolfs Consulting und Verwaltungs-GmbH (RCV)

TrialACTIVE NOT RECRUITING
Jun 2014Clinical Registry Investigating Bardet-Biedl Syndrome

Marshfield Clinic Research Foundation

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

1

Total programs

1

Open now

Patient Assistance Programs1

IMCIVREE

Rhythm Pharmaceuticals, Inc

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Approved Treatments

1 FDA-approved

IMCIVREE

(SETMELANOTIDE)standard

Rhythm Pharmaceuticals, Inc

Melanocortin 4 Receptor Agonist [EPC]

12.1 Mechanism of Action Setmelanotide is an MC4 receptor agonist with 20-fold less activity at the melanocortin 3 (MC3) and melanocortin 1 (MC1) rece...

FDA label ↗

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Clinical Trial Landscape

2 active trials
2Unknown
2Total recruiting
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Recent News & Research

No recent news articles indexed yet for Bardet-Biedl syndrome.
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Specialist Network

Top 6 by expertise

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