Late-onset retinal degeneration

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ORPHA:67042OMIM:605670H35.5
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1FDA treatments1Active trials10Specialists8Treatment centers

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

Late-onset retinal degeneration (L-ORD) is a rare inherited eye disease that slowly damages the retina — the light-sensitive layer at the back of the eye. The retina is responsible for capturing images and sending them to the brain, so when it breaks down, vision becomes progressively worse over time. L-ORD is sometimes called 'late-onset retinal dystrophy' and is caused by changes in a gene called C1QTNF5 (also known as CTRP5). The disease usually does not cause noticeable problems until a person reaches their 50s or 60s, which is why it is called 'late-onset.' The most common early sign is difficulty seeing in dim light or at night, a problem called night blindness. Over time, people may also develop abnormal deposits under the retina, thinning of the retinal tissue, and eventually loss of central vision. Some people also develop abnormal blood vessel growth under the retina, similar to what happens in age-related macular degeneration (AMD). Because L-ORD looks a lot like AMD, it is sometimes misdiagnosed. Currently, there is no cure for L-ORD, and no treatments have been specifically approved for this condition. Management focuses on monitoring vision changes, using low-vision aids, and in some cases treating complications like abnormal blood vessel growth. Research is ongoing, and gene therapy approaches are being explored. Regular follow-up with a retinal specialist is very important for people living with this condition.

Also known as:

Key symptoms:

Difficulty seeing in low light or at night (night blindness)Gradual loss of central visionBlurry or distorted visionAbnormal deposits under the retina visible on eye examsThinning of the retinal tissue over timeAbnormal blood vessel growth under the retinaSensitivity to bright lightLoss of color vision in later stagesReduced ability to read or recognize facesSlow adaptation when moving between light and dark environments

Clinical phenotype terms (22)— hover any for plain English
Abnormal best corrected visual acuity testHP:0030534Chorioretinal atrophyHP:0000533Macular degenerationHP:0000608Severely reduced visual acuityHP:0001141Patchy atrophy of the retinal pigment epitheliumHP:0007791Choroidal neovascularizationHP:0011506DrusenHP:0011510Subretinal depositsHP:0031528TritanomalyHP:0000552Red-green dyschromatopsiaHP:0000642Iris atrophyHP:0001089Atrophic fundus lesionHP:0001099Abnormal anterior eye segment morphologyHP:0004328Macular atrophyHP:0007401Abnormal suspensory ligament of lens morphologyHP:0012628Iris transillumination defectHP:0012805Peripapillary atrophyHP:0500087
Inheritance

Autosomal dominant

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

Age of Onset

Late onset

Begins later in life, typically after age 50

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

3 events
Jan 2023The Relationship Between Myonectin Concentration and the Course of ST-segment Elevation Myocardial Infarction

Michał Jaśkiewicz

TrialRECRUITING
Oct 2022Clinical Trial of Low-dose Radiation Therapy in Patients With Knee Osteoarthritis (LoRD-KNeA Trial)

Seoul National University Hospital — NA

TrialACTIVE NOT RECRUITING
Jul 2006

Normocarb HF: FDA approved

Use as a replacement solution in Continuous Renal Replacement Therapy (CRRT) to replace water and to correct electrolytes and acid-base imbalances in adults and children

FDAcompleted

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

Treatments

1 available

Normocarb HF

bicarbonate infusate· Dialysis Solutions, Inc.Orphan Drug

Use as a replacement solution in Continuous Renal Replacement Therapy (CRRT) to replace water and to correct electrolytes and acid-base imbalances in adults and children

Clinical Trials

1 recruitingView all trials with filters →
N/A1 trial
Clinical Trial of Low-dose Radiation Therapy in Patients With Knee Osteoarthritis (LoRD-KNeA Trial)
N/A
Active
PI: Byoung Hyuck Kim, MD, PhD (SMG-SNU Boramae Medical Center) · Sites: Seoul; Seoul +1 more · Age: 5085 yrs

Specialists

10 foundView all specialists →
FP
Flavio Mantelli, MD, PhD
Specialist
PI on 4 active trials
DP
Daniel X Hammer, Ph.D.
Specialist
PI on 1 active trial
LP
Laura Andres-Martin, PhD
Specialist
PI on 1 active trial
DP
David Liao, MD, PhD
Specialist
PI on 1 active trial
FP
Fnu Ruchi, Ph.D.
Specialist
PI on 1 active trial
SB
Shyamanga Borooah
SAN DIEGO, CA
Specialist
3 Late-onset retinal degeneration publications
LL
Leonardo Lando
CA
Specialist
2 Late-onset retinal degeneration publications
RM
Roly Megaw
Specialist
2 Late-onset retinal degeneration publications
BD
Baljean Dhillon
Specialist
2 Late-onset retinal degeneration publications
AB
Andrew C Browning
Specialist
2 Late-onset retinal degeneration publications

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 Late-onset retinal degeneration.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Late-onset retinal degeneration

Disease timeline:

New recruiting trial: The Relationship Between Myonectin Concentration and the Course of ST-segment Elevation Myocardial Infarction

A new clinical trial is recruiting patients for Late-onset retinal degeneration

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.Has my diagnosis been confirmed with genetic testing for the C1QTNF5 gene?,How quickly do you expect my vision to change, and what signs should prompt me to call you urgently?,Am I at risk for the complication of abnormal blood vessel growth, and how will we monitor for it?,Should my children or siblings be tested for this condition?,Are there any clinical trials for L-ORD that I might be eligible to join?,What low vision aids or rehabilitation services do you recommend for me right now?,Are there any vitamins, supplements, or lifestyle changes that might help protect my remaining vision?

Common questions about Late-onset retinal degeneration

What is Late-onset retinal degeneration?

Late-onset retinal degeneration (L-ORD) is a rare inherited eye disease that slowly damages the retina — the light-sensitive layer at the back of the eye. The retina is responsible for capturing images and sending them to the brain, so when it breaks down, vision becomes progressively worse over time. L-ORD is sometimes called 'late-onset retinal dystrophy' and is caused by changes in a gene called C1QTNF5 (also known as CTRP5). The disease usually does not cause noticeable problems until a person reaches their 50s or 60s, which is why it is called 'late-onset.' The most common early sign is

How is Late-onset retinal degeneration inherited?

Late-onset retinal degeneration follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Late-onset retinal degeneration typically begin?

Typical onset of Late-onset retinal degeneration is late onset. Age of onset can vary across affected individuals.

Are there clinical trials for Late-onset retinal degeneration?

Yes — 1 recruiting clinical trial is currently listed for Late-onset retinal degeneration on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Late-onset retinal degeneration?

10 specialists and care centers treating Late-onset retinal degeneration are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Late-onset retinal degeneration?

1 patient support program are currently tracked on UniteRare for Late-onset retinal degeneration. See the treatments and support programs sections for copay assistance, eligibility, and contact details.