Zellweger-like syndrome without peroxisomal anomalies

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:50812Q87.8
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

Zellweger-like syndrome without peroxisomal anomalies is a very rare genetic condition that closely resembles Zellweger syndrome in its symptoms but is different in one important way: the tiny cell structures called peroxisomes appear normal under testing. In classic Zellweger syndrome, peroxisomes are broken or missing. In this condition, peroxisomes look fine, yet the person still shows many of the same serious problems. This makes diagnosis especially challenging. The condition mainly affects the brain, liver, and overall development. Babies born with this syndrome often have very low muscle tone (called hypotonia), making them feel floppy. They may have seizures, feeding difficulties, and abnormal facial features. The brain may not develop properly, leading to significant intellectual disability and developmental delays. The liver can also be affected, causing abnormal liver function. There is currently no cure for this condition. Treatment focuses on managing symptoms, supporting development, and improving quality of life. This includes anti-seizure medications, feeding support, physical therapy, and regular monitoring of organ function. Because so few cases have been reported, much about this disease is still being studied and understood.

Also known as:

Key symptoms:

Very low muscle tone (floppy baby appearance)Seizures or epilepsyIntellectual disabilityDelayed development of motor and cognitive skillsAbnormal facial features (such as a high forehead, wide-set eyes, or a flat face)Liver problems or abnormal liver functionFeeding difficulties in infancyPoor weight gain and growthHearing lossVision problems

Clinical phenotype terms (22)— hover any for plain English
Brittle hairHP:0002299
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Zellweger-like syndrome without peroxisomal anomalies.

View clinical trials →

No actively recruiting trials found for Zellweger-like syndrome without peroxisomal anomalies at this time.

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

Search ClinicalTrials.gov ↗Join the Zellweger-like syndrome without peroxisomal anomalies community →

No specialists are currently listed for Zellweger-like syndrome without peroxisomal anomalies.

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 Zellweger-like syndrome without peroxisomal anomalies.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Zellweger-like syndrome without peroxisomal anomaliesForum →

No community posts yet. Be the first to share your experience with Zellweger-like syndrome without peroxisomal anomalies.

Start the conversation →

Latest news about Zellweger-like syndrome without peroxisomal anomalies

No recent news articles for Zellweger-like syndrome without peroxisomal anomalies.

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 genetic tests should we do to confirm this diagnosis and find the specific gene involved?,What specialists should be part of our care team, and how often should we see each one?,What is the best plan for managing my child's seizures, and what should we do in an emergency?,Are there any clinical trials or research studies we could participate in?,What therapies (physical, occupational, speech) would benefit my child most right now?,How should we monitor my child's liver health over time?,What support resources are available for our family as caregivers?

Common questions about Zellweger-like syndrome without peroxisomal anomalies

What is Zellweger-like syndrome without peroxisomal anomalies?

Zellweger-like syndrome without peroxisomal anomalies is a very rare genetic condition that closely resembles Zellweger syndrome in its symptoms but is different in one important way: the tiny cell structures called peroxisomes appear normal under testing. In classic Zellweger syndrome, peroxisomes are broken or missing. In this condition, peroxisomes look fine, yet the person still shows many of the same serious problems. This makes diagnosis especially challenging. The condition mainly affects the brain, liver, and overall development. Babies born with this syndrome often have very low musc

How is Zellweger-like syndrome without peroxisomal anomalies inherited?

Zellweger-like syndrome without peroxisomal anomalies follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Zellweger-like syndrome without peroxisomal anomalies typically begin?

Typical onset of Zellweger-like syndrome without peroxisomal anomalies is neonatal. Age of onset can vary across affected individuals.