Congenital muscular dystrophy with hyperlaxity

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Overview

Congenital muscular dystrophy with hyperlaxity (also called CMD with hyperlaxity or Ullrich-like congenital muscular dystrophy with hyperlaxity) is a rare inherited muscle disorder that is present from birth or early infancy. In this condition, babies are born with weak muscles and unusually loose (hyperlax or hypermobile) joints, meaning their joints can bend much further than normal. The combination of muscle weakness and joint looseness can make it harder for children to reach motor milestones like sitting, standing, and walking. The disease belongs to a group of conditions called congenital muscular dystrophies, which are characterized by muscle weakness and wasting that begins very early in life. Children with this form may also have soft, doughy skin, and some may develop joint contractures (stiffening of certain joints) over time, even though other joints remain overly flexible. Breathing difficulties can develop as the muscles that support the lungs may weaken over time. There is currently no cure for this condition. Treatment focuses on managing symptoms and improving quality of life. This includes physical therapy to maintain joint mobility and muscle strength, respiratory support if breathing becomes affected, and orthopedic interventions for contractures or spinal problems. A team of specialists typically works together to provide the best care. Research into the underlying genetic causes continues, and advances in gene therapy and other approaches offer hope for future treatments.

Also known as:

Key symptoms:

Muscle weakness present from birthUnusually loose or floppy joints (hyperlaxity)Low muscle tone (floppy baby)Delayed motor milestones such as sitting and walkingJoint contractures that develop over timeSoft or doughy skin textureBreathing difficultiesSpinal curvature (scoliosis or kyphosis)Difficulty feeding in infancyProgressive muscle wastingDifficulty with fine motor tasksFatigue and reduced endurance

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 Congenital muscular dystrophy with hyperlaxity.

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No actively recruiting trials found for Congenital muscular dystrophy with hyperlaxity at this time.

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No specialists are currently listed for Congenital muscular dystrophy with hyperlaxity.

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 Congenital muscular dystrophy with hyperlaxity.

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Community

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Caregiver Resources

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Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific genetic mutation is causing my child's condition, and what does it mean for their outlook?,How often should my child have breathing tests and respiratory monitoring?,What physical therapy program is best to maintain mobility and prevent contractures?,Are there any clinical trials or new treatments being studied for this condition?,What signs should I watch for that would require emergency medical attention?,How will this condition affect my child's ability to attend school and participate in activities?,Should other family members be tested for this genetic condition?

Common questions about Congenital muscular dystrophy with hyperlaxity

What is Congenital muscular dystrophy with hyperlaxity?

Congenital muscular dystrophy with hyperlaxity (also called CMD with hyperlaxity or Ullrich-like congenital muscular dystrophy with hyperlaxity) is a rare inherited muscle disorder that is present from birth or early infancy. In this condition, babies are born with weak muscles and unusually loose (hyperlax or hypermobile) joints, meaning their joints can bend much further than normal. The combination of muscle weakness and joint looseness can make it harder for children to reach motor milestones like sitting, standing, and walking. The disease belongs to a group of conditions called congenit

How is Congenital muscular dystrophy with hyperlaxity inherited?

Congenital muscular dystrophy with hyperlaxity follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Congenital muscular dystrophy with hyperlaxity typically begin?

Typical onset of Congenital muscular dystrophy with hyperlaxity is neonatal. Age of onset can vary across affected individuals.