Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome

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Overview

Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome is an extremely rare condition that affects multiple body systems, primarily involving the muscles, liver, and pregnancy-related fluid levels. The name describes its three hallmark features: muscular hypertrophy (abnormally enlarged muscles), hepatomegaly (an enlarged liver), and polyhydramnios (excess amniotic fluid surrounding the baby during pregnancy). This condition is typically identified before or shortly after birth due to these distinctive signs. Babies with this syndrome may appear unusually muscular at birth, and their liver may be noticeably enlarged on physical examination or imaging. The excess amniotic fluid during pregnancy can sometimes lead to premature delivery or complications during labor. Additional features may include other organ abnormalities and developmental concerns, though the full spectrum of the condition is still being understood due to its extreme rarity. Because so few cases have been described in the medical literature, the treatment landscape is largely supportive and symptom-based. There are no specific therapies or cures currently available. Management focuses on monitoring organ function, addressing complications as they arise, and providing supportive care. A team of specialists is typically needed to coordinate care for affected individuals.

Key symptoms:

Abnormally large or bulky muscles at birthEnlarged liverExcess amniotic fluid during pregnancyPremature birthBreathing difficulties at birthFeeding difficulties in newbornsAbnormal muscle tonePossible heart abnormalitiesPossible kidney problemsGrowth abnormalities

Clinical phenotype terms (14)— hover any for plain English
Retinal dysplasiaHP:0007973Type II lissencephalyHP:0007260Ocular anterior segment dysgenesisHP:0007700Macrocephaly at birthHP:0004488Occipital encephaloceleHP:0002085
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 Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome.

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No actively recruiting trials found for Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome at this time.

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No specialists are currently listed for Muscular hypertrophy-hepatomegaly-polyhydramnios 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 Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the current status of my child's liver function, and how often should it be monitored?,Are there any genetic tests that could help identify the exact cause of this condition?,What therapies or interventions can help support my child's development?,What warning signs should I watch for at home that would require emergency care?,Are there any clinical trials or research studies we could participate in?,How will this condition affect my child's growth and development over time?,Should other family members be tested or evaluated?

Common questions about Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome

What is Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome?

Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome is an extremely rare condition that affects multiple body systems, primarily involving the muscles, liver, and pregnancy-related fluid levels. The name describes its three hallmark features: muscular hypertrophy (abnormally enlarged muscles), hepatomegaly (an enlarged liver), and polyhydramnios (excess amniotic fluid surrounding the baby during pregnancy). This condition is typically identified before or shortly after birth due to these distinctive signs. Babies with this syndrome may appear unusually muscular at birth, and their liver

How is Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome inherited?

Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome typically begin?

Typical onset of Muscular hypertrophy-hepatomegaly-polyhydramnios syndrome is neonatal. Age of onset can vary across affected individuals.