Rare hereditary connective tissue disease

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ORPHA:619249
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17Specialists8Treatment centers

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

Rare hereditary connective tissue disease (Orphanet code 619249) is an extremely rare genetic condition that affects the body's connective tissue. Connective tissue is the material that holds together and supports many parts of the body, including skin, bones, joints, blood vessels, and internal organs. When connective tissue does not form or function properly, it can lead to problems in multiple body systems. Because this is an ultra-rare condition with very limited published information, the full range of symptoms and their severity is not yet well understood. Patients with hereditary connective tissue disorders may experience joint hypermobility (overly flexible joints), skin that is unusually stretchy or fragile, easy bruising, skeletal abnormalities, and problems with blood vessels or internal organs. The specific combination and severity of symptoms can vary widely from person to person. Treatment for this condition is currently focused on managing symptoms and preventing complications, as no cure exists. Care typically involves a team of specialists who work together to address the different body systems that may be affected. Physical therapy, pain management, and regular monitoring of the heart and blood vessels are common parts of the care plan. Research into the genetic basis of rare connective tissue diseases is ongoing, and new discoveries may lead to better treatments in the future.

Key symptoms:

Overly flexible or loose jointsSkin that stretches more than normalFragile skin that tears or bruises easilyChronic joint painJoint dislocations or subluxationsSkeletal abnormalitiesSlow wound healingMuscle weaknessFatigueProblems with blood vessels

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Rare hereditary connective tissue disease.

View clinical trials →

No actively recruiting trials found for Rare hereditary connective tissue disease at this time.

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

Search ClinicalTrials.gov ↗Join the Rare hereditary connective tissue disease community →

Specialists

17 foundView all specialists →
LP
Laurence Heidet, PHD
Specialist
PI on 1 active trial
AS
Ann (Annie) W Silk
Los Angeles, California
Specialist

Rare Disease Specialist

PI on 1 active trial
SA
Srivandana Akshintala
WASHINGTON, DC
Specialist
PI on 1 active trial
AM
Alessandro Pini, MD
San Donato Milanese, Lombardy
Specialist

Rare Disease Specialist

PI on 3 active trials
SP
Staci M Peron, Ph.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials
CM
Carlos R Ferreira Lopez, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 2 active trials
TM
Tammuella Chrisentery-Singleton, MD
Phoenix, Arizona
Specialist

Rare Disease Specialist

PI on 2 active trials
MM
Michael Recht, MD, PhD, MBA
CLEVELAND, OH
Specialist
PI on 1 active trial
MP
Marcelo Serra, PhD
Buenos Aires, Buenos Aires
Specialist

Rare Disease Specialist

PI on 1 active trial
MP
Maria Lourdes Posadas Martinez, PhD
Specialist
PI on 1 active trial
ZM
Zagorka Pejin, MD
Specialist
PI on 1 active trial
RP
Rosanna Cardani, PhD
San Donato Milanese, Milan
Specialist

Rare Disease 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 Rare hereditary connective tissue disease.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Rare hereditary connective tissue disease

No recent news articles for Rare hereditary connective tissue disease.

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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.What specific type of connective tissue disorder do I or my child have, and is the genetic cause known?,Which body systems should be monitored regularly, and how often?,Are there any activities or sports I should avoid to protect my joints and tissues?,What are the warning signs of a serious complication that would require emergency care?,Is genetic testing recommended for other family members?,Are there any clinical trials or research studies I could participate in?,What specialists should be part of my care team?

Common questions about Rare hereditary connective tissue disease

What is Rare hereditary connective tissue disease?

Rare hereditary connective tissue disease (Orphanet code 619249) is an extremely rare genetic condition that affects the body's connective tissue. Connective tissue is the material that holds together and supports many parts of the body, including skin, bones, joints, blood vessels, and internal organs. When connective tissue does not form or function properly, it can lead to problems in multiple body systems. Because this is an ultra-rare condition with very limited published information, the full range of symptoms and their severity is not yet well understood. Patients with hereditary conne

Which specialists treat Rare hereditary connective tissue disease?

17 specialists and care centers treating Rare hereditary connective tissue disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.