Cutis laxa-Marfanoid syndrome

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ORPHA:171719OMIM:614100Q87.8
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8Treatment centers

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Overview

Cutis laxa-Marfanoid syndrome is an extremely rare genetic condition that combines features of two different connective tissue disorders: cutis laxa and Marfan syndrome. Connective tissue acts like the body's glue, holding skin, bones, blood vessels, and organs together. When this tissue doesn't form properly, it can affect many parts of the body at once. People with this syndrome typically have loose, sagging skin that hangs in folds (cutis laxa) along with features seen in Marfan syndrome, such as a tall and thin body build, long limbs and fingers, joint hypermobility (overly flexible joints), and possible eye or heart problems. The skin may appear prematurely aged because it loses its elasticity. Some individuals may also experience developmental delays or intellectual disability. Because this condition is so rare, there is no specific cure. Treatment focuses on managing individual symptoms. This may include monitoring the heart and blood vessels for complications, eye exams, physical therapy for joint problems, and sometimes surgical procedures to address severely sagging skin. A team of specialists working together is usually needed to provide the best care.

Key symptoms:

Loose, sagging skin that lacks elasticityTall, thin body buildUnusually long arms, legs, and fingersOverly flexible or loose jointsAged-looking skin, even in young peoplePossible heart or blood vessel problemsEye problems such as nearsightedness or lens dislocationFlat feetCurved spine (scoliosis)Sunken or protruding chestPossible developmental delaysHerniasLung problems or emphysemaFacial features that may appear older than actual age

Clinical phenotype terms (8)— hover any for plain English
EmphysemaHP:0002097
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 Cutis laxa-Marfanoid syndrome.

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No actively recruiting trials found for Cutis laxa-Marfanoid syndrome at this time.

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No specialists are currently listed for Cutis laxa-Marfanoid syndrome.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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 Cutis laxa-Marfanoid syndrome.

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Community

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Latest news about Cutis laxa-Marfanoid syndrome

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Caregiver Resources

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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 genetic testing should be done to confirm the diagnosis?,How often should my heart and blood vessels be checked?,Are there any activities or sports that should be avoided?,What signs of complications should I watch for at home?,Should other family members be tested for this condition?,Are there any clinical trials or research studies we could participate in?,What specialists should be part of our care team, and how often should we see them?

Common questions about Cutis laxa-Marfanoid syndrome

What is Cutis laxa-Marfanoid syndrome?

Cutis laxa-Marfanoid syndrome is an extremely rare genetic condition that combines features of two different connective tissue disorders: cutis laxa and Marfan syndrome. Connective tissue acts like the body's glue, holding skin, bones, blood vessels, and organs together. When this tissue doesn't form properly, it can affect many parts of the body at once. People with this syndrome typically have loose, sagging skin that hangs in folds (cutis laxa) along with features seen in Marfan syndrome, such as a tall and thin body build, long limbs and fingers, joint hypermobility (overly flexible joint

How is Cutis laxa-Marfanoid syndrome inherited?

Cutis laxa-Marfanoid syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Cutis laxa-Marfanoid syndrome typically begin?

Typical onset of Cutis laxa-Marfanoid syndrome is neonatal. Age of onset can vary across affected individuals.