Bone dysplasia, lethal Holmgren type

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ORPHA:1842OMIM:211120Q77.8
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Overview

Bone dysplasia, lethal Holmgren type is an extremely rare skeletal disorder that affects how bones develop before birth. This condition is classified as a lethal bone dysplasia, meaning it is severe enough to cause death either before birth or shortly after. The disease was described by Holmgren and colleagues, and it belongs to a group of conditions where the skeleton does not form properly during fetal development. Babies affected by this condition typically show severely shortened limbs, an abnormally shaped chest that is often very narrow or small, and other skeletal abnormalities that can be detected on prenatal ultrasound. The small chest cavity restricts lung development, which leads to serious breathing difficulties that are usually not compatible with life. Other features may include abnormal curvature of the spine and unusual changes in the growth plates of bones. Because this condition is lethal, there are no curative treatments available. Medical care focuses on providing comfort and supportive measures. Genetic counseling is important for affected families to understand the risk of recurrence in future pregnancies. Due to the extreme rarity of this condition, much about its underlying genetic cause and precise mechanisms remains poorly understood.

Also known as:

Key symptoms:

Severely shortened arms and legsVery small or narrow chestUnderdeveloped lungsSevere breathing difficulties at birthAbnormal bone shape visible on X-rayShort statureAbnormal growth plates in bonesFlattened vertebrae in the spineReduced movement before birthExcess fluid around the baby before birth (hydrops)

Clinical phenotype terms (39)— hover any for plain English
Severe short-limb dwarfismHP:0008890Abnormal diaphysis morphologyHP:0000940Bell-shaped thoraxHP:0001591
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 Bone dysplasia, lethal Holmgren type.

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No specialists are currently listed for Bone dysplasia, lethal Holmgren type.

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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

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.How certain is this diagnosis, and could it be a different type of skeletal dysplasia?,Is genetic testing available that could help identify the exact cause?,What is the chance this could happen again in a future pregnancy?,What palliative care and comfort options are available for our baby?,Can you refer us to a genetic counselor to discuss family planning?,Are there any research studies or registries we could participate in?,What bereavement and mental health support services are available for our family?

Common questions about Bone dysplasia, lethal Holmgren type

What is Bone dysplasia, lethal Holmgren type?

Bone dysplasia, lethal Holmgren type is an extremely rare skeletal disorder that affects how bones develop before birth. This condition is classified as a lethal bone dysplasia, meaning it is severe enough to cause death either before birth or shortly after. The disease was described by Holmgren and colleagues, and it belongs to a group of conditions where the skeleton does not form properly during fetal development. Babies affected by this condition typically show severely shortened limbs, an abnormally shaped chest that is often very narrow or small, and other skeletal abnormalities that ca

How is Bone dysplasia, lethal Holmgren type inherited?

Bone dysplasia, lethal Holmgren type follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Bone dysplasia, lethal Holmgren type typically begin?

Typical onset of Bone dysplasia, lethal Holmgren type is neonatal. Age of onset can vary across affected individuals.