Autosomal dominant optic atrophy

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ORPHA:98672
Who is this for?
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2Active trials8Treatment centers

Where are you in your journey?

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

DOAADOA
Orphanet ↗NORD ↗

FDA & Trial Timeline

3 events
Sep 2025Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy

PYC Therapeutics — PHASE1, PHASE2

TrialRECRUITING
Oct 2024SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

PYC Therapeutics — PHASE1

TrialACTIVE NOT RECRUITING
Jan 2024Tolerance and Efficacy Nicotinamide (vitamin B3) in Dominant Optic Atrophy OPA1

University Hospital, Angers — PHASE2, PHASE3

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Autosomal dominant optic atrophy.

2 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

2 recruitingView all trials with filters →
Phase 11 trial
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
Phase 1
Active
PI: Sreenivasu Mudumba, PhD (PYC Therapeutics) · Sites: Sydney, New South Wales; East Melbourne, Victoria · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

View all specialists →

No specialists are currently listed for Autosomal dominant optic atrophy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center ↗

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center ↗

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program ↗

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site ↗

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site ↗

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site ↗

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine ↗

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program ↗

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Autosomal dominant optic atrophy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Autosomal dominant optic atrophy

Disease timeline:

New recruiting trial: Tolerance and Efficacy Nicotinamide (vitamin B3) in Dominant Optic Atrophy OPA1

A new clinical trial is recruiting patients for Autosomal dominant optic atrophy

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Autosomal dominant optic atrophy

Are there clinical trials for Autosomal dominant optic atrophy?

Yes — 2 recruiting clinical trials are currently listed for Autosomal dominant optic atrophy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.