Trichothiodystrophy

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ORPHA:33364OMIM:619691L67.8
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2Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Trichothiodystrophy (TTD) is a rare inherited condition that mainly affects the hair, skin, nails, and nervous system. The name comes from Greek words meaning 'sulfur-deficient hair' — one of the most recognizable features is brittle hair that breaks easily and has unusually low levels of sulfur. When viewed under a special microscope with polarized light, the hair shows a distinctive 'tiger tail' banding pattern, which is a key clue for diagnosis. TTD affects many parts of the body. People with TTD often have intellectual disability, delayed development, short stature, and problems with coordination. The skin may be dry and scaly (a condition called ichthyosis), and the nails can be brittle. Some people with TTD are also very sensitive to sunlight because their cells have trouble repairing DNA damage caused by ultraviolet (UV) rays. Other features can include a small head size (microcephaly), fertility problems, and a weakened immune system that makes infections more likely. The severity of TTD varies widely from person to person — some individuals have mild symptoms while others are more severely affected. There is currently no cure for TTD. Treatment focuses on managing symptoms, such as protecting the skin, supporting development through therapies, and preventing infections. A team of specialists is usually needed to provide the best care.

Key symptoms:

Brittle hair that breaks easilyHair with low sulfur content and a 'tiger tail' pattern under polarized lightDry, scaly skin (ichthyosis)Brittle nailsIntellectual disability or learning difficultiesDelayed development (walking, talking later than expected)Short statureSmall head size (microcephaly)Sensitivity to sunlight (in some forms)Poor coordination and balance problemsWeakened immune system leading to frequent infectionsFertility problems in some individualsCataracts (clouding of the eye lens) in some cases

Clinical phenotype terms (50)— hover any for plain English
Gonadal dysgenesisHP:0000133Bird-like faciesHP:0000320Retinal degenerationHP:0000546Macular degenerationHP:0000608
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 Trichothiodystrophy.

View clinical trials →

No actively recruiting trials found for Trichothiodystrophy at this time.

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

Search ClinicalTrials.gov ↗Join the Trichothiodystrophy community →

Specialists

2 foundView all specialists →
MM
Michael R Sargen, M.D.
PHILADELPHIA, PA
Specialist
PI on 2 active trials
PM
Peter Kang, MD
Minneapolis, Minnesota
Specialist

Rare Disease Specialist

PI on 2 active trials

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 Trichothiodystrophy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Trichothiodystrophy

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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.Which gene is causing TTD in my child, and what does that mean for their specific symptoms and outlook?,Does my child have the form of TTD with UV sensitivity, and how strictly do we need to protect them from sunlight?,What therapies (physical, occupational, speech) should my child start, and how often?,How often should my child be seen by each specialist, and what should we be monitoring over time?,Are there any clinical trials or research studies that my child might be eligible for?,What signs of infection or other complications should prompt an emergency visit?,Are other family members at risk, and should siblings or parents be tested?

Common questions about Trichothiodystrophy

What is Trichothiodystrophy?

Trichothiodystrophy (TTD) is a rare inherited condition that mainly affects the hair, skin, nails, and nervous system. The name comes from Greek words meaning 'sulfur-deficient hair' — one of the most recognizable features is brittle hair that breaks easily and has unusually low levels of sulfur. When viewed under a special microscope with polarized light, the hair shows a distinctive 'tiger tail' banding pattern, which is a key clue for diagnosis. TTD affects many parts of the body. People with TTD often have intellectual disability, delayed development, short stature, and problems with coor

How is Trichothiodystrophy inherited?

Trichothiodystrophy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Trichothiodystrophy typically begin?

Typical onset of Trichothiodystrophy is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Trichothiodystrophy?

2 specialists and care centers treating Trichothiodystrophy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.