Microcephalic primordial dwarfism, Dauber type

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ORPHA:319675OMIM:614851Q87.1
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Overview

Microcephalic primordial dwarfism, Dauber type, is an extremely rare genetic condition characterized by very short stature that begins before birth and a smaller-than-normal head size (microcephaly). The term 'primordial dwarfism' means that growth restriction starts very early in development, even before a baby is born, and continues after birth. Children and adults with this condition are significantly shorter than their peers, and their head circumference is notably smaller than average, though intellectual development may be relatively preserved compared to some other forms of primordial dwarfism. This condition is caused by changes (mutations) in a gene involved in growth signaling pathways. Because the condition affects overall body growth from the earliest stages of development, individuals tend to have proportionate short stature, meaning their body parts are small but generally in proportion to each other. Some individuals may also have mild facial differences and other subtle physical features. There is currently no cure for this condition. Treatment focuses on monitoring growth, managing any associated health concerns, and providing supportive care. Growth hormone therapy has been explored in some forms of primordial dwarfism, but its effectiveness in this specific type may be limited. Regular follow-up with specialists is important to track development and address any complications that may arise over time.

Key symptoms:

Very short stature present from birthSmaller-than-normal head size (microcephaly)Low birth weightShort body length at birthProportionate short stature throughout lifeDelayed growth milestonesMild facial differencesThin body buildSmall hands and feetPossible mild learning difficulties in some cases

Clinical phenotype terms (22)— hover any for plain English
Abnormal carpal morphologyHP:0001191Subglottic stenosisHP:0001607Madelung deformityHP:0003067Short middle phalanx of the 5th fingerHP:0004220Lumbar scoliosisHP:0004626Severe intrauterine growth retardationHP:0008846Severe postnatal growth retardationHP:0008850
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Microcephalic primordial dwarfism, Dauber type.

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No specialists are currently listed for Microcephalic primordial dwarfism, Dauber type.

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 Microcephalic primordial dwarfism, Dauber type.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific genetic change was found, and what does it mean for my child's health?,Is growth hormone therapy an option, and what are the expected benefits and risks?,What developmental milestones should we watch for, and when should we be concerned?,Are there any other health complications we should screen for regularly?,What early intervention or therapy services would benefit my child?,Is there a risk that future children could also have this condition?,Are there any clinical trials or research studies we could participate in?

Common questions about Microcephalic primordial dwarfism, Dauber type

What is Microcephalic primordial dwarfism, Dauber type?

Microcephalic primordial dwarfism, Dauber type, is an extremely rare genetic condition characterized by very short stature that begins before birth and a smaller-than-normal head size (microcephaly). The term 'primordial dwarfism' means that growth restriction starts very early in development, even before a baby is born, and continues after birth. Children and adults with this condition are significantly shorter than their peers, and their head circumference is notably smaller than average, though intellectual development may be relatively preserved compared to some other forms of primordial d

How is Microcephalic primordial dwarfism, Dauber type inherited?

Microcephalic primordial dwarfism, Dauber type follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Microcephalic primordial dwarfism, Dauber type typically begin?

Typical onset of Microcephalic primordial dwarfism, Dauber type is neonatal. Age of onset can vary across affected individuals.