Progressive bulbar paralysis of childhood

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Overview

Progressive bulbar paralysis of childhood, also known as Fazio-Londe disease, is a rare and serious condition that affects the nerves controlling the muscles of the face, mouth, throat, and voice box. It belongs to a group of diseases called motor neuron diseases, where the nerve cells that send signals to muscles gradually stop working. The disease typically begins in childhood or early adolescence and slowly gets worse over time. The main nerves affected are called the lower cranial nerves. When these nerves stop working properly, the muscles they control become weak and eventually stop functioning. This leads to problems with swallowing, speaking, chewing, and breathing. Children may first notice changes in their voice, difficulty eating, or facial weakness before other symptoms appear. There is currently no cure for Fazio-Londe disease. Treatment focuses on managing symptoms, keeping the child as comfortable as possible, and supporting breathing and nutrition as the disease progresses. A team of specialists is usually needed to provide the best care. Research into this condition is ongoing, and some cases have been linked to changes in the gene that controls how the body uses riboflavin (vitamin B2), which has opened the door to potential treatment in certain patients.

Also known as:

Key symptoms:

Weakness of the facial musclesDifficulty swallowing (dysphagia)Changes in voice, such as hoarseness or a nasal-sounding voiceDifficulty chewing foodWeakness of the tongueBreathing problems due to weak throat and airway musclesDroolingSlurred or unclear speechWeakness spreading to the arms and legs in some casesUnintentional weight loss due to difficulty eating

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Progressive bulbar paralysis of childhood.

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No actively recruiting trials found for Progressive bulbar paralysis of childhood at this time.

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No specialists are currently listed for Progressive bulbar paralysis of childhood.

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 Progressive bulbar paralysis of childhood.

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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.Has genetic testing confirmed which gene is causing my child's disease, and does my child qualify for riboflavin therapy?,How quickly do you expect the disease to progress, and what signs should I watch for that mean things are getting worse?,When should we start thinking about breathing support, and what options are available?,What is the best way to keep my child eating safely, and when might a feeding tube become necessary?,Are there any clinical trials or research studies my child could participate in?,What specialists should be part of my child's care team, and how often should we see each one?,What support services are available for our family, including mental health support and respite care?

Common questions about Progressive bulbar paralysis of childhood

What is Progressive bulbar paralysis of childhood?

Progressive bulbar paralysis of childhood, also known as Fazio-Londe disease, is a rare and serious condition that affects the nerves controlling the muscles of the face, mouth, throat, and voice box. It belongs to a group of diseases called motor neuron diseases, where the nerve cells that send signals to muscles gradually stop working. The disease typically begins in childhood or early adolescence and slowly gets worse over time. The main nerves affected are called the lower cranial nerves. When these nerves stop working properly, the muscles they control become weak and eventually stop fun

How is Progressive bulbar paralysis of childhood inherited?

Progressive bulbar paralysis of childhood follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Progressive bulbar paralysis of childhood typically begin?

Typical onset of Progressive bulbar paralysis of childhood is childhood. Age of onset can vary across affected individuals.