Retinal macular dystrophy type 2

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ORPHA:319640OMIM:608051H35.5
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8Specialists8Treatment centers

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

Retinal macular dystrophy type 2 (also known as MCDR2 or North Carolina-like macular dystrophy) is a rare inherited eye condition that affects the macula, which is the central part of the retina responsible for sharp, detailed vision. The retina is the light-sensitive tissue at the back of the eye, and the macula is its most important area for tasks like reading, driving, and recognizing faces. In this condition, the macula gradually deteriorates over time, leading to progressive loss of central vision. People with this disease typically notice difficulty with fine visual tasks such as reading small print or seeing details clearly. Peripheral (side) vision is usually preserved, meaning that complete blindness is uncommon. The severity can vary significantly between affected individuals, even within the same family. Some people may have relatively mild vision changes, while others experience more significant central vision loss. Currently, there is no cure or specific treatment that can reverse or halt the progression of retinal macular dystrophy type 2. Management focuses on regular eye monitoring, use of low-vision aids such as magnifying devices, and adaptive strategies to help maintain quality of life. Research into gene therapy and other approaches for inherited retinal diseases is ongoing and may offer future treatment possibilities.

Also known as:

Key symptoms:

Gradual loss of central visionBlurred vision, especially for fine detailsDifficulty reading small printTrouble recognizing facesDistorted vision where straight lines appear wavyReduced color perception in the central visual fieldDark or blank spots in the center of visionDifficulty seeing in low-light conditionsPreserved peripheral (side) visionVariable severity between family members

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Apr 2026Efficacy of Faricimab in Patients With Subretinal Hyper-reflective Material

Biobizkaia Health Research Institute — PHASE4

TrialNOT YET RECRUITING
Mar 2025Investigating the Metabolic and Lipidomic Profiles That Are Associated With Varying Grades of Diabetic Maculopathy and Retinopathy in South Wales

Hywel Dda Health Board

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Retinal macular dystrophy type 2.

View clinical trials →

No actively recruiting trials found for Retinal macular dystrophy type 2 at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Retinal macular dystrophy type 2 community →

Specialists

8 foundView all specialists →
KM
Kai Zhang, MD
Specialist
PI on 2 active trials
LP
Laura Andres-Martin, PhD
Specialist
PI on 1 active trial
FF
Francis WB Sanders, MB BChir PhD FRCOphth
Specialist
PI on 1 active trial
KM
Karim Hammamji, MD
Specialist
PI on 1 active trial
RM
Ronald Klein, MD, MPH
Specialist
PI on 1 active trial
AM
Alberto L Gil, MD
Specialist
PI on 1 active trial1 Retinal macular dystrophy type 2 publication
CP
Ciro Costagliola, Professor
Specialist
PI on 1 active trial
RM
Roberto dell'Omo, MD
Specialist
PI on 1 active trial

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 Retinal macular dystrophy type 2.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Retinal macular dystrophy type 2

Disease timeline:

New recruiting trial: Investigating the Metabolic and Lipidomic Profiles That Are Associated With Varying Grades of Diabetic Maculopathy and Retinopathy in South Wales

A new clinical trial is recruiting patients for Retinal macular dystrophy type 2

New recruiting trial: Evaluation of NeoRetina Artificial Intelligence Algorithm for the Screening of Diabetic Retinopathy at the CHUM

A new clinical trial is recruiting patients for Retinal macular dystrophy type 2

New recruiting trial: MS-553 in Diabetic Retinopathy Patients With Central Involved Macular Edema

A new clinical trial is recruiting patients for Retinal macular dystrophy type 2

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.

Questions for your doctor

Bring these to your next appointment

  • Q1.How advanced is the macular dystrophy in my eyes right now, and what is the expected rate of progression?,Are there any clinical trials or emerging treatments for PROM1-related retinal dystrophy that I might be eligible for?,What low-vision aids or rehabilitation services would you recommend for my current level of vision?,How often should I have follow-up eye exams, and what specific tests will be done?,Should my family members be tested for this genetic condition?,Are there any signs of complications like abnormal blood vessel growth that I should watch for?,Can you refer me to a genetic counselor to discuss inheritance and family planning?

Common questions about Retinal macular dystrophy type 2

What is Retinal macular dystrophy type 2?

Retinal macular dystrophy type 2 (also known as MCDR2 or North Carolina-like macular dystrophy) is a rare inherited eye condition that affects the macula, which is the central part of the retina responsible for sharp, detailed vision. The retina is the light-sensitive tissue at the back of the eye, and the macula is its most important area for tasks like reading, driving, and recognizing faces. In this condition, the macula gradually deteriorates over time, leading to progressive loss of central vision. People with this disease typically notice difficulty with fine visual tasks such as readin

How is Retinal macular dystrophy type 2 inherited?

Retinal macular dystrophy type 2 follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Which specialists treat Retinal macular dystrophy type 2?

8 specialists and care centers treating Retinal macular dystrophy type 2 are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.