Autosomal ichthyosis syndrome with fatal disease course

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Overview

Autosomal ichthyosis syndrome with fatal disease course is an extremely rare and severe skin disorder that belongs to a group of conditions called ichthyoses. In these conditions, the skin cannot form a proper protective barrier, leading to widespread thickening, scaling, and cracking of the skin. This particular form follows an autosomal pattern of inheritance and is distinguished from other ichthyoses by its severity and ultimately fatal outcome, typically occurring in the newborn period or early infancy. Babies born with this condition often present at birth or very shortly after with dramatically abnormal skin. The skin may appear tight, thick, and covered in plate-like scales, sometimes resembling a collodion membrane (a shiny, tight film covering the body). Because the skin barrier is so severely compromised, affected infants face life-threatening complications including massive fluid loss, inability to regulate body temperature, overwhelming infections, and breathing difficulties due to restricted chest wall movement. Feeding problems and failure to thrive are also common. There is currently no cure for this condition. Treatment is entirely supportive and focuses on keeping the skin moisturized, preventing and treating infections, maintaining body temperature, ensuring adequate nutrition, and providing intensive neonatal care. Despite aggressive medical support, the disease course is fatal, usually within the first days to weeks of life. Genetic counseling is critically important for affected families to understand recurrence risks and options for future pregnancies.

Key symptoms:

Thick, plate-like scaling of the skin over the entire bodyExtremely tight skin at birthCollodion membrane (shiny, tight film covering the skin at birth)Severe cracking and fissuring of the skinRedness of the skinDifficulty breathing due to tight chest skinProblems with feeding and swallowingInability to regulate body temperatureSevere dehydration from fluid loss through the skinRecurrent skin infectionsRestricted movement of arms and legsSwelling of hands and feetAbnormal facial features due to tight skinFailure to gain weight and growEyelids and lips may be turned outward

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal ichthyosis syndrome with fatal disease course.

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No actively recruiting trials found for Autosomal ichthyosis syndrome with fatal disease course at this time.

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No specialists are currently listed for Autosomal ichthyosis syndrome with fatal disease course.

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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 Autosomal ichthyosis syndrome with fatal disease course.

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the specific genetic cause of my baby's condition, and has genetic testing been done?,What can be done to keep my baby comfortable?,Can we be connected with a palliative care team?,What is the chance this could happen again in a future pregnancy?,Are there prenatal testing options available for future pregnancies?,Can you refer us to a genetic counselor?,What emotional support services are available for our family?

Common questions about Autosomal ichthyosis syndrome with fatal disease course

What is Autosomal ichthyosis syndrome with fatal disease course?

Autosomal ichthyosis syndrome with fatal disease course is an extremely rare and severe skin disorder that belongs to a group of conditions called ichthyoses. In these conditions, the skin cannot form a proper protective barrier, leading to widespread thickening, scaling, and cracking of the skin. This particular form follows an autosomal pattern of inheritance and is distinguished from other ichthyoses by its severity and ultimately fatal outcome, typically occurring in the newborn period or early infancy. Babies born with this condition often present at birth or very shortly after with dram

At what age does Autosomal ichthyosis syndrome with fatal disease course typically begin?

Typical onset of Autosomal ichthyosis syndrome with fatal disease course is neonatal. Age of onset can vary across affected individuals.