Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly

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ORPHA:402364OMIM:613668Q04.3
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Overview

Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly is an extremely rare genetic brain disorder that affects infants from birth or very early in life. In this condition, the brain — including both the cerebrum (the large upper part) and the cerebellum (the smaller lower part responsible for coordination) — progressively shrinks or fails to grow properly after birth. This leads to a head size that becomes increasingly smaller than expected over time, a feature called progressive microcephaly. Affected babies typically appear normal at birth or shortly after, but within the first months of life they begin to show signs of severe developmental delay. They may have difficulty feeding, fail to reach milestones like holding their head up or sitting, and develop seizures. Movement problems, including stiffness or floppiness of the limbs, are common. Vision and hearing may also be affected. The condition is caused by mutations in the CASK gene or other genes involved in brain development, depending on the specific genetic subtype. There is currently no cure for this condition. Treatment focuses on managing symptoms such as seizures, feeding difficulties, and movement problems. A team of specialists including neurologists, geneticists, and therapists work together to provide supportive care and improve quality of life. The prognosis is generally severe, with significant intellectual disability and physical limitations.

Key symptoms:

Head size that becomes progressively smaller after birthSevere developmental delaySeizures or epilepsyDifficulty feeding or swallowingMuscle stiffness or spasticityLow muscle tone (floppiness)Inability to sit, stand, or walk independentlyIntellectual disabilityPoor or absent speech developmentAbnormal eye movementsVision problemsIrritability or excessive cryingShrinking of brain tissue visible on imagingPoor head controlBreathing difficulties

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly.

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No actively recruiting trials found for Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly at this time.

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No specialists are currently listed for Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly.

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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 Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly.

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Community

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Latest news about Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly

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Caregiver Resources

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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 specific gene mutation is causing my child's condition, and what does it mean for their outlook?,What seizure medications are best for my child, and what side effects should I watch for?,Should we consider a feeding tube, and when is the right time?,What therapies can help my child be as comfortable as possible?,Are there any clinical trials or research studies my child might be eligible for?,Should other family members be tested for the genetic change?,Can you connect us with palliative care or family support services?

Common questions about Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly

What is Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly?

Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly is an extremely rare genetic brain disorder that affects infants from birth or very early in life. In this condition, the brain — including both the cerebrum (the large upper part) and the cerebellum (the smaller lower part responsible for coordination) — progressively shrinks or fails to grow properly after birth. This leads to a head size that becomes increasingly smaller than expected over time, a feature called progressive microcephaly. Affected babies typically appear normal at birth or shortly after, but w

At what age does Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly typically begin?

Typical onset of Infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly is infantile. Age of onset can vary across affected individuals.