Pinsky-Di George-Harley syndrome

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:2895
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

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

Overview

Pinsky-Di George-Harley syndrome is an extremely rare genetic condition that was first described in the medical literature in the 1960s. It is also sometimes referred to as Pinsky syndrome. This syndrome primarily affects males and is characterized by a combination of intellectual disability, distinctive facial features, and skeletal abnormalities. Affected individuals may have short stature, unusual facial characteristics such as a broad or flat nose, widely spaced eyes, and abnormalities of the hands and feet. Some patients also experience hearing loss and genital abnormalities such as underdeveloped genitalia (hypogonadism). The condition is thought to follow an X-linked recessive pattern of inheritance, meaning it mainly affects boys, while mothers can be carriers without showing symptoms. Because this syndrome is so rare, with only a handful of cases reported in the medical literature, there is limited information about the full range of symptoms and the best approaches to treatment. Management is generally supportive and focuses on addressing individual symptoms, such as special education services for intellectual disability, orthopedic care for skeletal problems, and hearing aids if hearing loss is present. There is currently no cure or disease-specific therapy available.

Also known as:

Key symptoms:

Intellectual disabilityShort statureDistinctive facial featuresBroad or flat noseWidely spaced eyesAbnormalities of the hands and feetHearing lossUnderdeveloped genitaliaSkeletal abnormalitiesDelayed developmentLow muscle toneSpeech delay

Inheritance

X-linked recessive

Carried on the X chromosome; typically affects males more than females

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Pinsky-Di George-Harley syndrome.

View clinical trials →

No actively recruiting trials found for Pinsky-Di George-Harley syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Pinsky-Di George-Harley syndrome community →

No specialists are currently listed for Pinsky-Di George-Harley syndrome.

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 Pinsky-Di George-Harley syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Pinsky-Di George-Harley syndromeForum →

No community posts yet. Be the first to share your experience with Pinsky-Di George-Harley syndrome.

Start the conversation →

Latest news about Pinsky-Di George-Harley syndrome

No recent news articles for Pinsky-Di George-Harley syndrome.

Follow this condition to be notified when news becomes available.

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.What specific symptoms should we watch for as our child grows?,Is genetic testing available that could help confirm the diagnosis or identify the exact gene involved?,What early intervention services do you recommend for our child's development?,How often should hearing and vision be checked?,Are there any skeletal problems that need to be monitored or treated?,Should we see an endocrinologist about genital or growth concerns?,Are there any other family members who should be tested as possible carriers?

Common questions about Pinsky-Di George-Harley syndrome

What is Pinsky-Di George-Harley syndrome?

Pinsky-Di George-Harley syndrome is an extremely rare genetic condition that was first described in the medical literature in the 1960s. It is also sometimes referred to as Pinsky syndrome. This syndrome primarily affects males and is characterized by a combination of intellectual disability, distinctive facial features, and skeletal abnormalities. Affected individuals may have short stature, unusual facial characteristics such as a broad or flat nose, widely spaced eyes, and abnormalities of the hands and feet. Some patients also experience hearing loss and genital abnormalities such as under

How is Pinsky-Di George-Harley syndrome inherited?

Pinsky-Di George-Harley syndrome follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Pinsky-Di George-Harley syndrome typically begin?

Typical onset of Pinsky-Di George-Harley syndrome is neonatal. Age of onset can vary across affected individuals.