Hypohidrotic ectodermal dysplasia

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ORPHA:238468OMIM:129490Q82.4
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13Specialists8Treatment centers

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

Hypohidrotic ectodermal dysplasia (HED), also known as Christ-Siemens-Touraine syndrome, is a genetic condition that affects the development of structures that come from the ectoderm — the outer layer of the embryo. This means the skin, hair, nails, teeth, and sweat glands do not develop normally. The hallmark features of HED are reduced ability to sweat (hypohidrosis), sparse and thin hair (hypotrichosis), and missing or abnormally shaped teeth (hypodontia). Because the body cannot sweat properly, people with HED are at serious risk of overheating (hyperthermia), which can be life-threatening, especially in infants and young children. Beyond the three main features, individuals may also have dry skin, dark circles around the eyes, a flattened bridge of the nose, and prominent forehead. The condition is present from birth, though it may not be recognized immediately. Some babies with HED experience unexplained high fevers because they cannot cool themselves through sweating. There is currently no cure for HED, and treatment focuses on managing symptoms. This includes avoiding overheating, using cooling devices, dental treatments such as dentures or implants, and skin moisturizers. Recent research into a protein replacement therapy (ER004) given before birth has shown promise in clinical trials, but it is not yet widely available. With proper management and awareness of heat-related risks, most people with HED can live full and productive lives.

Also known as:

Key symptoms:

Reduced or absent sweatingThin, sparse, light-colored hairMissing teeth or cone-shaped teethDry skinOverheating and heat intoleranceFrequent respiratory infectionsDry eyesDry nose with crustingDark circles around the eyesFlattened bridge of the noseProminent foreheadThin or absent eyebrows and eyelashesPeeling skin in newbornsDifficulty feeding in infancyHoarse or raspy voice

Clinical phenotype terms (31)— hover any for plain English
Slow-growing hairHP:0002217Abnormal abdominal wall morphologyHP:0004298
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 ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Jan 2021HED-Start: Evaluating a Positive Skills Intervention for Patients New on Haemodialysis

Nanyang Technological University — NA

TrialRECRUITING
Sep 2015Correlation of the Arrhythmia Mechanism and Substrate to Ablate Persistent Atrial Fibrillation Study

Ottawa Heart Institute Research Corporation — NA

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Hypohidrotic ectodermal dysplasia.

View clinical trials →

No actively recruiting trials found for Hypohidrotic ectodermal dysplasia at this time.

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

Search ClinicalTrials.gov ↗Join the Hypohidrotic ectodermal dysplasia community →

Specialists

13 foundView all specialists →
DM
Dorothy K Grange, MD
SAINT LOUIS, MO
Specialist
PI on 6 active trials
MG
Marlène Guiraud
Specialist
PI on 1 active trial1 Hypohidrotic ectodermal dysplasia publication
JM
James Maynard, MD
Specialist
PI on 2 active trials
KP
Kenneth Huttner, MD, PhD
WALTHAM, MA
Specialist
PI on 2 active trials
OP
Ophir Klein, MD, PhD
WEST HOLLYWOOD, CA
Specialist
PI on 2 active trials
EP
Encarna Navarro, MD, PhD
Specialist
PI on 1 active trial
CC
Carrie Milliard, CGC
Specialist
PI on 1 active trial
SP
Smail Hadj-Rabia, MD, PhD
Specialist
PI on 1 active trial
BP
Brigitte Fauroux, MD, PhD
Specialist
PI on 1 active trial
LM
Lucie Griffon, MD
Specialist
PI on 1 active trial1 Hypohidrotic ectodermal dysplasia publication
LP
Lisa Moscoso, MD, PhD
SAINT LOUIS, MO
Specialist
PI on 1 active trial

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 Hypohidrotic ectodermal dysplasia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Hypohidrotic ectodermal dysplasia

Disease timeline:

New recruiting trial: HED-Start: Evaluating a Positive Skills Intervention for Patients New on Haemodialysis

A new clinical trial is recruiting patients for Hypohidrotic ectodermal dysplasia

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.How severe is my child's sweating ability, and what specific cooling strategies do you recommend?,When should we start dental treatment, and what options are available for young children?,Are there any clinical trials for new treatments that my child might be eligible for?,What school accommodations should we request to keep my child safe?,How can we tell the difference between a normal fever from illness and dangerous overheating?,Should other family members be tested to see if they carry the gene change?,What specialists should we see regularly, and how often?

Common questions about Hypohidrotic ectodermal dysplasia

What is Hypohidrotic ectodermal dysplasia?

Hypohidrotic ectodermal dysplasia (HED), also known as Christ-Siemens-Touraine syndrome, is a genetic condition that affects the development of structures that come from the ectoderm — the outer layer of the embryo. This means the skin, hair, nails, teeth, and sweat glands do not develop normally. The hallmark features of HED are reduced ability to sweat (hypohidrosis), sparse and thin hair (hypotrichosis), and missing or abnormally shaped teeth (hypodontia). Because the body cannot sweat properly, people with HED are at serious risk of overheating (hyperthermia), which can be life-threatening

At what age does Hypohidrotic ectodermal dysplasia typically begin?

Typical onset of Hypohidrotic ectodermal dysplasia is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Hypohidrotic ectodermal dysplasia?

13 specialists and care centers treating Hypohidrotic ectodermal dysplasia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.