Hereditary vascular retinopathy

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ORPHA:71291
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32Specialists8Treatment centers

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

Hereditary vascular retinopathy (HVR) is a rare inherited condition that affects the tiny blood vessels in the retina — the light-sensitive layer at the back of the eye. Over time, these blood vessels become damaged, leading to problems with vision that can gradually worsen. The condition is sometimes called hereditary endotheliopathy, retinopathy, nephropathy, and stroke (HERNS) when it also affects the kidneys and brain, or it may be referred to as cerebroretinal vasculopathy (CRV) or hereditary systemic angiopathy. These names reflect the fact that in some people, the disease goes beyond the eyes and can affect blood vessels in the brain and kidneys as well. The main symptoms involve progressive vision loss, which can include blurry vision, blind spots, and difficulty seeing in low light. Some people also experience strokes or stroke-like episodes, migraines, and kidney problems, depending on how widely the disease affects the body's blood vessels. Symptoms usually begin in adulthood, though the age of onset can vary. There is currently no cure for hereditary vascular retinopathy. Treatment focuses on managing symptoms, monitoring vision and organ function, and reducing the risk of complications like stroke. Regular follow-up with eye specialists, neurologists, and kidney doctors is important. Research into this condition is ongoing, and genetic testing can help confirm the diagnosis and guide family planning decisions.

Also known as:

Key symptoms:

Gradual loss of visionBlurry or distorted visionBlind spots in the visual fieldDifficulty seeing in dim lightFloaters or flashes of lightStroke or stroke-like episodesMigraine headaches, sometimes with visual disturbancesWeakness or numbness on one side of the bodyMemory or thinking problemsProtein in the urine (a sign of kidney involvement)High blood pressure related to kidney diseaseFatigue

Inheritance

Autosomal dominant

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

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

3 events
May 2024A Study to Evaluate the Efficacy and Safety of Tarcocimab Tedromer Compared With Sham Treatment in Participants With Diabetic Retinopathy (DR)

Kodiak Sciences Inc — PHASE3

TrialACTIVE NOT RECRUITING
Jan 2022Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD

AbbVie — PHASE3

TrialRECRUITING
Dec 2020Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD

AbbVie — PHASE2, PHASE3

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Hereditary vascular retinopathy.

View clinical trials →

No actively recruiting trials found for Hereditary vascular retinopathy at this time.

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

Search ClinicalTrials.gov ↗Join the Hereditary vascular retinopathy community →

Specialists

Showing 25 of 32View all specialists →
MM
Michio Hirano, MD
NEW YORK, NY
Specialist
PI on 10 active trials
PM
Pablo Velazquez-Martin, MD
Specialist
PI on 8 active trials
RM
Robin S Roberts, MTech
Specialist
PI on 2 active trials
IM
Ina Koetter, MD
Specialist
PI on 1 active trial1 Hereditary vascular retinopathy publication
HM
Haresh Kirpalani, MD, MSc
PHILADELPHIA, PA
Specialist
PI on 2 active trials
KM
Kim E. Nichols, MD
Memphis, Tennessee
Specialist

Rare Disease Specialist

PI on 2 active trials
TD
Thomas Klopstock, Prof. Dr.
Specialist
PI on 3 active trials
PM
Peter A Campochiaro, MD
BALTIMORE, MD
Specialist
PI on 9 active trials
CM
Chadwick Prodromos, MD
GLENVIEW, IL
Specialist
PI on 11 active trials
MD
Michelangelo Mancuso, Prof. Dr.
Specialist
PI on 1 active trial
MP
Michel PAQUES
Specialist
PI on 4 active trials2 Hereditary vascular retinopathy publications
XM
Xi Chen, MD
Specialist
PI on 1 active trial
CP
Chi Hornik, PharmD
DURHAM, NC
Specialist
PI on 1 active trial
DR
Dr. Niaz Abdur Rahman
Specialist
PI on 1 active trial
MP
Morris Blajchman, PhD
Specialist
PI on 1 active trial
RM
Robin K Whyte, MD
Specialist
PI on 1 active trial
EM
Elizabeth Asztalos, MD, MSc
Specialist
PI on 1 active trial
CM
Chad Andersen, MD
ST GEORGE, UT
Specialist
PI on 1 active trial
NM
Nancy Heddle, MSc
Specialist
PI on 1 active trial
CM
Christopher D Riemann, MD
CINCINNATI, OH
Specialist
PI on 1 active trial
DE
Dr. Alaa Abdelkarim Mohammed, MRCPUK Endocirnology
Specialist
PI on 1 active trial
FD
Frank G. Holz, MD, DPhil
Specialist
PI on 1 active trial
RP
Robert Bhisitkul, MD, Ph.D.
SAN FRANCISCO, CA
Specialist
PI on 1 active trial
DO
Dr. Passant Sayed Saif, PhD ophthalmology
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 Hereditary vascular retinopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Hereditary vascular retinopathyForum →

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Latest news about Hereditary vascular retinopathy

Disease timeline:

New recruiting trial: A Study to Collect Data on the Use of Eylea in Babies Born Too Early Who Have a Condition of the Eye Where Blood Vessels Grow Abnormally in the Retina (Retinopathy of Prematurity)

A new clinical trial is recruiting patients for Hereditary vascular retinopathy

New recruiting trial: Imaging Retinal Vasculature in Infant Eyes

A new clinical trial is recruiting patients for Hereditary vascular retinopathy

New recruiting trial: Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD

A new clinical trial is recruiting patients for Hereditary vascular retinopathy

New recruiting trial: Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies

A new clinical trial is recruiting patients for Hereditary vascular retinopathy

New recruiting trial: A Efficacy and Safety Study of Ranibizumab 10mg/ml Injection (Incepta) in Patients With Diabetic Macular Edema

A new clinical trial is recruiting patients for Hereditary vascular retinopathy

New recruiting trial: Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD

A new clinical trial is recruiting patients for Hereditary vascular retinopathy

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.Which organs are affected in my case, and how will you monitor each of them?,Should my children or siblings be tested for the TREX1 gene mutation?,Are there any clinical trials or research studies I could join?,What can I do to reduce my risk of stroke?,How quickly is my vision likely to change, and what vision aids might help me?,Are there any medications or lifestyle changes that could slow the progression of this disease?,What signs should prompt me to go to the emergency room immediately?

Common questions about Hereditary vascular retinopathy

What is Hereditary vascular retinopathy?

Hereditary vascular retinopathy (HVR) is a rare inherited condition that affects the tiny blood vessels in the retina — the light-sensitive layer at the back of the eye. Over time, these blood vessels become damaged, leading to problems with vision that can gradually worsen. The condition is sometimes called hereditary endotheliopathy, retinopathy, nephropathy, and stroke (HERNS) when it also affects the kidneys and brain, or it may be referred to as cerebroretinal vasculopathy (CRV) or hereditary systemic angiopathy. These names reflect the fact that in some people, the disease goes beyond th

How is Hereditary vascular retinopathy inherited?

Hereditary vascular retinopathy follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hereditary vascular retinopathy typically begin?

Typical onset of Hereditary vascular retinopathy is adult. Age of onset can vary across affected individuals.

Which specialists treat Hereditary vascular retinopathy?

25 specialists and care centers treating Hereditary vascular retinopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.