CANDLE syndrome

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ORPHA:325004
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18Specialists8Treatment centers

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

CANDLE syndrome stands for Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated temperature. It is also known as Nakajo-Nishimura syndrome, JMP syndrome, or proteasome-associated autoinflammatory syndrome (PRAAS). This is a very rare genetic condition that affects the immune system, causing it to be overactive from a very young age. The disease belongs to a group of conditions called proteasome-associated autoinflammatory syndromes. The hallmark of CANDLE syndrome is recurring fevers that begin in infancy, along with distinctive skin rashes that look like red or violet ring-shaped patches. Over time, children develop lipodystrophy, which means they lose fat tissue under the skin, especially in the face and arms, giving a thin or wasted appearance. The condition also causes joint pain and swelling, muscle wasting, and delayed growth. Many patients develop an enlarged liver, and blood tests often show signs of ongoing inflammation, including anemia. There is currently no cure for CANDLE syndrome. Treatment focuses on controlling inflammation and managing symptoms. JAK inhibitors, particularly baricitinib, have shown significant promise in clinical trials and are used to reduce inflammation and fevers. Before JAK inhibitors became available, treatment options were very limited, as the disease often did not respond well to standard anti-inflammatory medications or immunosuppressive drugs. Early diagnosis and treatment are important to prevent long-term damage to organs and tissues.

Also known as:

Key symptoms:

Recurring fevers starting in infancyRed or purple ring-shaped skin rashesLoss of fat tissue under the skin (lipodystrophy), especially in the faceDelayed growth and short statureJoint pain and swellingMuscle wasting and weaknessEnlarged liverSwollen eyelids, especially with puffy or violet discolorationAnemia (low red blood cell count)Elevated inflammatory markers in blood testsThickened or hardened skin in some areasCalcium deposits in body tissues (calcifications)Fatigue and low energyClubbing of fingers

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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for CANDLE syndrome.

View clinical trials →

No actively recruiting trials found for CANDLE syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the CANDLE syndrome community →

Specialists

18 foundView all specialists →
RG
Raphaela Goldbach-Mansky
BETHESDA, MD
Specialist
2 CANDLE syndrome publications
MB
Manfred Boehm
Specialist
2 CANDLE syndrome publications
GC
Guibin Chen
Specialist
2 CANDLE syndrome publications
CW
Clayton W Winkler
COLUMBIA, SC
Specialist
1 CANDLE syndrome publication
JS
Joseph Snow
Specialist
1 CANDLE syndrome publication
CF
Christopher Friend
CHARLOTTESVILLE, VA
Specialist
1 CANDLE syndrome publication
FB
Farzana Bhuyan
Specialist
1 CANDLE syndrome publication
SA
Sara Alehashemi
BETHESDA, MD
Specialist
2 CANDLE syndrome publications
EB
Eric Bohrnsen
Specialist
1 CANDLE syndrome publication
KW
Katie Williams
Specialist
1 CANDLE syndrome publication
SF
Simote T Foliaki
Specialist
1 CANDLE syndrome publication
GC
Gwendolyn Cooper
PEARLAND, TX
Specialist
1 CANDLE syndrome publication
AT
Audrey Thurm
BOSTON, MA
Specialist
1 CANDLE syndrome publication
LJ
Lisa Joseph
Specialist
1 CANDLE syndrome publication
BS
Benjamin Schwarz
NEW YORK, NY
Specialist
1 CANDLE syndrome publication
KM
Karin R Laursen, MSc
OAKLAND, CA
Specialist
PI on 1 active trial
TP
Torben Sigsgaard, Prof.
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 CANDLE syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about CANDLE syndrome

No recent news articles for CANDLE syndrome.

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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.Is my child eligible for treatment with baricitinib or other JAK inhibitors?,Are there any clinical trials currently enrolling patients with CANDLE syndrome?,How often will my child need blood tests and monitoring?,What signs should prompt me to seek emergency care?,How can we support my child's growth and nutrition?,What are the long-term risks of the medications being used?,Should other family members be tested for the genetic mutations?

Common questions about CANDLE syndrome

What is CANDLE syndrome?

CANDLE syndrome stands for Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated temperature. It is also known as Nakajo-Nishimura syndrome, JMP syndrome, or proteasome-associated autoinflammatory syndrome (PRAAS). This is a very rare genetic condition that affects the immune system, causing it to be overactive from a very young age. The disease belongs to a group of conditions called proteasome-associated autoinflammatory syndromes. The hallmark of CANDLE syndrome is recurring fevers that begin in infancy, along with distinctive skin rashes that look like red or violet rin

How is CANDLE syndrome inherited?

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

At what age does CANDLE syndrome typically begin?

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

Which specialists treat CANDLE syndrome?

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