Lethal congenital contracture syndrome

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ORPHA:294965
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24Specialists8Treatment centers

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Overview

Lethal congenital contracture syndrome (LCCS) is a group of very rare and severe inherited conditions that affect a baby before birth. Babies with this condition develop fixed joint positions (called contractures), meaning their arms, legs, and other joints cannot move normally. The condition is caused by problems with the nerves and muscles that control movement during fetal development. Because the baby cannot move properly in the womb, the joints become stiff and locked in place. This is sometimes called fetal akinesia, meaning a lack of fetal movement. There are several subtypes of LCCS (types 1 through 11 and others), each linked to different genes. Common features across the subtypes include severe joint contractures present at birth, underdeveloped muscles, and often underdeveloped lungs. Many affected babies also have a very small jaw (micrognathia), excess fluid around the body (hydrops fetalis), and reduced amniotic fluid or excess amniotic fluid during pregnancy. Sadly, this condition is almost always fatal either before birth or shortly after delivery, primarily because the lungs do not develop enough to support breathing. There is currently no cure or effective treatment for LCCS. Management is focused on supportive and palliative care. Genetic counseling is very important for affected families, especially for understanding the risk in future pregnancies. Prenatal diagnosis through ultrasound and genetic testing can help identify the condition early in pregnancy.

Also known as:

Key symptoms:

Joints locked in fixed positions at birth (contractures)Little or no movement of the baby before birthSeverely underdeveloped musclesUnderdeveloped lungsVery small jawExcess fluid buildup around the baby's body (hydrops)Abnormal amounts of amniotic fluid during pregnancyThin or fragile bonesRounded or puffy facial appearanceShort neck with extra skin foldsAbnormal positioning of fingers and toesSmall body size for gestational ageInability to breathe independently at birth

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 ↗

FDA & Trial Timeline

1 event
Jan 2015A Study of Zenocutuzumab (MCLA-128) in Patients With Solid Tumors Harboring an NRG1 Fusion (eNRGy)

Partner Therapeutics, Inc. — PHASE2

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Lethal congenital contracture syndrome.

View clinical trials →

No actively recruiting trials found for Lethal congenital contracture syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Lethal congenital contracture syndrome community →

Specialists

24 foundView all specialists →
SB
Sonja Bläuer
Specialist
1 Lethal congenital contracture syndrome publication
CG
Christina A Gurnett
SAINT LOUIS, MO
Specialist
1 Lethal congenital contracture syndrome publication
ZS
Zoltan Szabo
KEALAKEKUA, HI
Specialist
1 Lethal congenital contracture syndrome publication
JW
Julia Whittle
BOXBOROUGH, MA
Specialist
1 Lethal congenital contracture syndrome publication
IM
Ilkka Miinalainen
Specialist
1 Lethal congenital contracture syndrome publication
AJ
Aaron Johnson
Specialist
1 Lethal congenital contracture syndrome publication
MD
Matthew B Dobbs
Specialist
1 Lethal congenital contracture syndrome publication
TZ
Tomáš Zárybnický
Specialist
1 Lethal congenital contracture syndrome publication
SL
Sonja Lindfors
Specialist
1 Lethal congenital contracture syndrome publication
SM
Saana Metso
Specialist
1 Lethal congenital contracture syndrome publication
JK
Julia Koivula
Specialist
1 Lethal congenital contracture syndrome publication
RV
Rasmus Valtonen
Specialist
1 Lethal congenital contracture syndrome publication
MT
Mikko Tulppo
Specialist
1 Lethal congenital contracture syndrome publication
JM
Johanna Magga
Specialist
1 Lethal congenital contracture syndrome publication
SS
Samu Saarimäki
Specialist
1 Lethal congenital contracture syndrome publication
BP
BioNTech Responsible Person
Specialist
PI on 18 active trials
AM
Alison Schram, MD
NEW YORK, NY
Specialist
PI on 1 active trial1 Lethal congenital contracture syndrome publication
EM
Ernesto Wasserman, MD
Specialist
PI on 1 active trial
KM
Keedy L Vicki, MD
Specialist
PI on 1 active trial
KM
Kwok Kin Wong, MD
Specialist
PI on 1 active trial
CM
Crystal Denlinger, MD
PHILADELPHIA, PA
Specialist
PI on 2 active trials
VM
Victor Moyo, MD
FARMINGTON, CT
Specialist
PI on 4 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 Lethal congenital contracture syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Lethal congenital contracture syndrome

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

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Family & Caregiver Grants

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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 subtype of lethal congenital contracture syndrome does our baby have, and which gene is involved?,What is the chance this could happen again in a future pregnancy?,Can we do genetic testing before or during a future pregnancy to check for this condition?,Are there palliative care services available to help us and our baby be comfortable?,Should other family members be tested to see if they are carriers?,Can you refer us to a genetic counselor who specializes in this type of condition?,What bereavement and psychological support services are available for our family?

Common questions about Lethal congenital contracture syndrome

What is Lethal congenital contracture syndrome?

Lethal congenital contracture syndrome (LCCS) is a group of very rare and severe inherited conditions that affect a baby before birth. Babies with this condition develop fixed joint positions (called contractures), meaning their arms, legs, and other joints cannot move normally. The condition is caused by problems with the nerves and muscles that control movement during fetal development. Because the baby cannot move properly in the womb, the joints become stiff and locked in place. This is sometimes called fetal akinesia, meaning a lack of fetal movement. There are several subtypes of LCCS (

How is Lethal congenital contracture syndrome inherited?

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

At what age does Lethal congenital contracture syndrome typically begin?

Typical onset of Lethal congenital contracture syndrome is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Lethal congenital contracture syndrome?

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