Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome

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ORPHA:500135OMIM:236500Q87.8
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Overview

Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome (sometimes abbreviated as MARCH syndrome) is an extremely rare and severe condition that affects brain development, kidney formation, and the environment around the baby before birth. The name describes the main features: abnormal brain cells with multiple nuclei (multinucleated neurons), little or no amniotic fluid surrounding the baby during pregnancy (anhydramnios), poorly formed kidneys (renal dysplasia), an underdeveloped part of the brain called the cerebellum (cerebellar hypoplasia), and a condition where most of the brain's cerebral hemispheres are replaced by fluid-filled sacs (hydranencephaly). This condition is typically detected before or at birth. The combination of severe brain malformations and kidney problems makes this one of the most serious rare diseases known. Babies affected by this syndrome face profound neurological challenges and life-threatening complications from the very start of life. The kidneys may not work properly, and the brain structure is severely disrupted, affecting nearly all basic functions. There is currently no cure for this syndrome, and treatment focuses entirely on comfort and supportive care. Management involves a team of specialists working together to address symptoms and support both the child and family. Because this condition is so rare, research is still in very early stages, and most knowledge comes from a small number of reported cases.

Also known as:

Key symptoms:

Little or no amniotic fluid around the baby during pregnancySeverely underdeveloped or absent brain tissue, replaced by fluid-filled spacesUnderdeveloped cerebellum (the part of the brain that controls balance and coordination)Poorly formed or non-functioning kidneysAbnormal brain cells with multiple nuclei seen under a microscopeSevere intellectual disabilityLittle to no voluntary movementDifficulty breathing at birthSeizuresFeeding difficultiesAbsent or very limited response to surroundings

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 ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome.

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No actively recruiting trials found for Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome community →

No specialists are currently listed for Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly 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 Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome.

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Community

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Latest news about Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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 is the likely prognosis for my child, and what should we expect in the coming days and weeks?,Which gene is responsible in our child's case, and what does that mean for future pregnancies?,What supportive treatments are available to keep our baby comfortable?,Should we pursue whole exome or whole genome sequencing, and how long will results take?,What palliative care services are available to us, and how do we access them?,Are there any research studies or registries we should know about for this condition?,What support resources exist for families going through this diagnosis?

Common questions about Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome

What is Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome?

Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome (sometimes abbreviated as MARCH syndrome) is an extremely rare and severe condition that affects brain development, kidney formation, and the environment around the baby before birth. The name describes the main features: abnormal brain cells with multiple nuclei (multinucleated neurons), little or no amniotic fluid surrounding the baby during pregnancy (anhydramnios), poorly formed kidneys (renal dysplasia), an underdeveloped part of the brain called the cerebellum (cerebellar hypoplasia), and a

How is Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome inherited?

Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome typically begin?

Typical onset of Multinucleated neurons-anhydramnios-renal dysplasia-cerebellar hypoplasia-hydranencephaly syndrome is neonatal. Age of onset can vary across affected individuals.