Achromatopsia Advocacy Hub | UniteRare

At a Glance

Live from database

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Regulatory Watchboard

5 events

Mar 2026RINGERS: New indication approved

FDAcompleted

Jul 2025Natural History Study of Inherited Retinal Diseases

Zhongmou Therapeutics

TrialRECRUITING

Aug 2017Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

Beacon Therapeutics — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Apr 2016Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

Beacon Therapeutics — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Nov 2015Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia

STZ eyetrial — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

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

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

Total programs

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Patient Assistance Programs1

NAGLAZYME

BioMarin Pharmaceutical Inc.

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Clinical Trial Landscape

4 active trials

1Unknown

3PHASE1, PHASE2

4Total recruiting

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Recent News & Research

No recent news articles indexed yet for Achromatopsia.
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Specialist Network

Top 6 by expertise

David Jacobs, MD, MBA

Applied Genetics Technologies Corporation

Score 92 trials

Brian P Brooks, M.D.

National Eye Institute (NEI)

📍 Bethesda, Maryland

Score 88 trials

James Bainbridge, M.D

Chief Investigator

📍 GREENWOOD VILLAGE, CO

Score 81 trial29 pubs

James Bainbridge, Prof, M.D

Chief Investigator

📍 GREENWOOD VILLAGE, CO

Score 82 trials

Dominik Fischer, Prof.

University Hospital Tuebingen, Center for Ophthalmology

Score 42 trials

Yin Shen

Renmin Hospital of Wuhan University

Score 31 trial331 pubs

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