Lethal congenital contracture syndrome type 2

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ORPHA:137776OMIM:607598Q68.8
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3Specialists8Treatment centers

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

Lethal congenital contracture syndrome type 2 (LCCS2), also known as multiple contracture syndrome, Israeli Bedouin type, is an extremely rare and severe autosomal recessive disorder characterized by multiple joint contractures (arthrogryposis), markedly reduced fetal movements, and early lethality. The condition is caused by mutations in the ERBB3 gene, which encodes a member of the epidermal growth factor receptor family of receptor tyrosine kinases. This gene plays a critical role in the development and survival of motor neurons and Schwann cells during embryonic development. Affected infants typically present at birth with severe arthrogryposis multiplex congenita, including contractures of multiple joints, particularly the limbs. Additional features include micrognathia (small jaw), pulmonary hypoplasia (underdeveloped lungs), pterygia (webbing of the skin across joints), and marked anterior horn cell degeneration in the spinal cord, reflecting a profound loss of lower motor neurons. Fetal akinesia (lack of movement) is a hallmark feature detectable during pregnancy. The condition is invariably lethal, with affected infants either stillborn or dying shortly after birth due to respiratory failure related to pulmonary hypoplasia and neurological compromise. LCCS2 was originally described in consanguineous Israeli Bedouin families. There is currently no curative treatment or disease-modifying therapy available for this condition. Management is limited to supportive and palliative care. Genetic counseling is essential for affected families, and prenatal diagnosis may be possible through molecular testing when the familial mutation is known. The condition belongs to a group of lethal congenital contracture syndromes (LCCS types 1–11) that share overlapping features of fetal akinesia and arthrogryposis but are caused by mutations in different genes.

Also known as:

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 ↗

FDA & Trial Timeline

3 events
Mar 2024Study of JK07 in Patients With Chronic Heart Failure

Salubris Biotherapeutics Inc — PHASE2

TrialRECRUITING
Feb 2024Study of an Anti-HER3 Antibody, HMBD-001, With Docetaxel +/- Cetuximab in Advanced Squamous Non-small Cell Lung Cancers, and HMBD-001 + Cetuximab in Advanced Squamous Cell Cancers

Hummingbird Bioscience — PHASE1, PHASE2

TrialRECRUITING
Oct 2017Neratinib and Everolimus, Palbociclib, or Trametinib in Treating Participants With Refractory and Advanced or Metastatic Solid Tumors With EGFR Mutation/Amplification, HER2 Mutation/Amplification, or HER3/4 Mutation or KRAS Mutation

M.D. Anderson Cancer Center — PHASE1

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 type 2.

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No actively recruiting trials found for Lethal congenital contracture syndrome type 2 at this time.

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Specialists

3 foundView all specialists →
GL
Global Clinical Leader
Specialist
PI on 44 active trials4 Lethal congenital contracture syndrome type 2 publications
SP
Sarina A Piha-Paul
Houston, Texas
Specialist

Rare Disease Specialist

PI on 2 active trials2 Lethal congenital contracture syndrome type 2 publications
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 type 2.

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Community

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Latest news about Lethal congenital contracture syndrome type 2

Disease timeline:

New recruiting trial: Study of an Anti-HER3 Antibody, HMBD-001, With Docetaxel +/- Cetuximab in Advanced Squamous Non-small Cell Lung Cancers, and HMBD-001 + Cetuximab in Advanced Squamous Cell Cancers

A new clinical trial is recruiting patients for Lethal congenital contracture syndrome type 2

New recruiting trial: Study of JK07 in Patients With Chronic Heart Failure

A new clinical trial is recruiting patients for Lethal congenital contracture syndrome type 2

Caregiver Resources

NORD Caregiver Resources

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Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Lethal congenital contracture syndrome type 2

What is Lethal congenital contracture syndrome type 2?

Lethal congenital contracture syndrome type 2 (LCCS2), also known as multiple contracture syndrome, Israeli Bedouin type, is an extremely rare and severe autosomal recessive disorder characterized by multiple joint contractures (arthrogryposis), markedly reduced fetal movements, and early lethality. The condition is caused by mutations in the ERBB3 gene, which encodes a member of the epidermal growth factor receptor family of receptor tyrosine kinases. This gene plays a critical role in the development and survival of motor neurons and Schwann cells during embryonic development. Affected infa

How is Lethal congenital contracture syndrome type 2 inherited?

Lethal congenital contracture syndrome type 2 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 type 2 typically begin?

Typical onset of Lethal congenital contracture syndrome type 2 is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Lethal congenital contracture syndrome type 2?

3 specialists and care centers treating Lethal congenital contracture syndrome type 2 are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.