OBSOLETE: Isolated optic nerve hypoplasia/aplasia

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ORPHA:137902
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Overview

Isolated optic nerve hypoplasia (ONH) or aplasia is a condition where one or both optic nerves — the nerves that carry visual information from the eyes to the brain — are underdeveloped (hypoplasia) or, in rare cases, completely absent (aplasia). This condition is present from birth. The term 'isolated' means it occurs on its own, without being part of a larger syndrome such as septo-optic dysplasia (de Morsier syndrome), which also involves brain and hormonal abnormalities. The main effect of this condition is reduced vision, which can range from mild visual impairment to complete blindness in the affected eye or eyes. When only one eye is involved, the other eye may have normal or near-normal vision. When both eyes are affected, vision loss tends to be more significant. Children with this condition may show involuntary eye movements (nystagmus), a wandering or misaligned eye (strabismus), and difficulty tracking objects. Note that this Orphanet entry is marked as 'OBSOLETE,' meaning it may have been reclassified or merged into a broader diagnostic category. Patients and families should discuss the most current classification with their doctor. There is currently no cure for optic nerve hypoplasia or aplasia. Treatment focuses on maximizing remaining vision through corrective lenses, patching therapy for amblyopia (lazy eye), and vision rehabilitation services. Early intervention with low-vision aids and educational support can significantly improve a child's development and quality of life.

Key symptoms:

Reduced vision in one or both eyesSmall or pale optic disc seen on eye examInvoluntary eye movements (nystagmus)Misaligned or wandering eyes (strabismus)Difficulty tracking objects with the eyesPoor depth perceptionReduced peripheral visionSensitivity to light in some casesDifficulty seeing in dim lightingOne pupil that reacts differently to light than the other

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for OBSOLETE: Isolated optic nerve hypoplasia/aplasia.

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No actively recruiting trials found for OBSOLETE: Isolated optic nerve hypoplasia/aplasia at this time.

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No specialists are currently listed for OBSOLETE: Isolated optic nerve hypoplasia/aplasia.

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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 OBSOLETE: Isolated optic nerve hypoplasia/aplasia.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.Is my child's optic nerve hypoplasia affecting one eye or both, and how severe is it?,Should we get an MRI to check for any brain or pituitary gland abnormalities?,Does my child need hormone testing to rule out associated endocrine problems?,Would genetic testing be helpful for our family, and what genes would be tested?,What vision rehabilitation services and early intervention programs are available?,How often should my child have eye exams going forward?,Are there any clinical trials or emerging treatments we should know about?

Common questions about OBSOLETE: Isolated optic nerve hypoplasia/aplasia

What is OBSOLETE: Isolated optic nerve hypoplasia/aplasia?

Isolated optic nerve hypoplasia (ONH) or aplasia is a condition where one or both optic nerves — the nerves that carry visual information from the eyes to the brain — are underdeveloped (hypoplasia) or, in rare cases, completely absent (aplasia). This condition is present from birth. The term 'isolated' means it occurs on its own, without being part of a larger syndrome such as septo-optic dysplasia (de Morsier syndrome), which also involves brain and hormonal abnormalities. The main effect of this condition is reduced vision, which can range from mild visual impairment to complete blindness

At what age does OBSOLETE: Isolated optic nerve hypoplasia/aplasia typically begin?

Typical onset of OBSOLETE: Isolated optic nerve hypoplasia/aplasia is neonatal. Age of onset can vary across affected individuals.