X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability

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ORPHA:480880OMIM:300968Q87.8
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8Treatment centers

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Overview

X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability is a very rare genetic condition that mainly affects females. It is sometimes referred to by its Orphanet code ORPHA:480880. The condition is caused by a change (variant) in a gene located on the X chromosome, and for reasons that are not yet fully understood, it appears to cause symptoms primarily in females rather than males. This condition affects several parts of the body at once. The most noticeable features include a distinctive facial appearance, being shorter than average for age, blockage of the nasal passages at the back of the nose (called choanal atresia), and varying degrees of intellectual disability or learning difficulties. Choanal atresia can cause serious breathing problems, especially in newborns, and often needs surgical correction early in life. There is currently no cure for this condition. Treatment focuses on managing individual symptoms. This may include surgery to open the nasal passages, educational support for learning difficulties, and therapies such as speech, occupational, and physical therapy. A team of different specialists is usually needed to provide the best care. Early diagnosis and intervention can make a meaningful difference in quality of life.

Also known as:

Key symptoms:

Distinctive facial features such as widely spaced eyes, a flat nasal bridge, or other unusual facial proportionsShort stature — being noticeably shorter than peers of the same ageChoanal atresia — a blockage at the back of the nasal passages that can make breathing difficultIntellectual disability or learning difficulties ranging from mild to moderateDelayed speech and language developmentFeeding difficulties, especially in infancyBreathing problems in newborns due to nasal blockagePossible hearing difficulties

Clinical phenotype terms (50)— hover any for plain English
Abnormal cortical gyrationHP:0002536Depigmentation/hyperpigmentation of skinHP:0007483Postaxial polydactylyHP:0100259
Inheritance

X-linked dominant

Carried on the X chromosome; a single copy can cause the condition

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability.

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No actively recruiting trials found for X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability at this time.

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Search ClinicalTrials.gov ↗Join the X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability community →

No specialists are currently listed for X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability.

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 X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability

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Caregiver Resources

NORD Caregiver Resources

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Family & Caregiver Grants

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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 gene is causing this condition in my child, and what does that mean for other family members?,Does my child need surgery for the nasal blockage, and when should it happen?,What therapies — speech, occupational, physical — should we start, and how soon?,What kind of educational support will my child need, and how do we access it?,Are there any other health problems we should watch for as my child grows?,Should other family members, including siblings, be tested?,Are there any clinical trials or research studies we could participate in?

Common questions about X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability

What is X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability?

X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability is a very rare genetic condition that mainly affects females. It is sometimes referred to by its Orphanet code ORPHA:480880. The condition is caused by a change (variant) in a gene located on the X chromosome, and for reasons that are not yet fully understood, it appears to cause symptoms primarily in females rather than males. This condition affects several parts of the body at once. The most noticeable features include a distinctive facial appearance, being shorter than average for age, block

How is X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability inherited?

X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability follows a x-linked dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability typically begin?

Typical onset of X-linked female restricted facial dysmorphism-short stature-choanal atresia-intellectual disability is neonatal. Age of onset can vary across affected individuals.