Salla disease

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ORPHA:309334OMIM:604369E77.8
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1Specialists8Treatment centers

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

Salla disease, also known as free sialic acid storage disease or sialuria Finnish type, is a rare inherited metabolic disorder that belongs to the group of lysosomal storage diseases. It is caused by a problem with how the body handles a sugar molecule called sialic acid (also known as N-acetylneuraminic acid). Normally, sialic acid is transported out of tiny compartments in cells called lysosomes after it has been used. In Salla disease, this transport system does not work properly, so sialic acid builds up inside the lysosomes, especially in the brain and other organs. This buildup damages cells over time. The disease typically becomes noticeable in infancy or early childhood. Affected children usually show delayed development, low muscle tone (hypotonia), and difficulty with movement and coordination (ataxia). Over time, most individuals develop intellectual disability that ranges from moderate to severe. Many children learn to walk, though often later than expected, and some develop the ability to speak a few words. Seizures can occur in some patients. The disease progresses slowly compared to the more severe form of sialic acid storage disease known as infantile sialic acid storage disease (ISSD). There is currently no cure for Salla disease. Treatment focuses on managing symptoms and supporting the individual's quality of life. Physical therapy, occupational therapy, speech therapy, and seizure management are the main approaches. Research into potential therapies is ongoing, but no disease-modifying treatments are currently available.

Key symptoms:

Delayed motor development (late sitting, late walking)Intellectual disability (moderate to severe)Low muscle tone (floppiness) in infancyDifficulty with balance and coordination (ataxia)Speech delay or limited speechInvoluntary rapid eye movements (nystagmus)Seizures or epilepsySpasticity (stiff muscles) that develops over timeCoarse facial features in some individualsShort statureThickened skinEnlarged liver or spleen in some casesDifficulty with fine motor skillsBehavioral challenges

Inheritance

Autosomal recessive

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

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Salla disease.

View clinical trials →

No actively recruiting trials found for Salla disease at this time.

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

Search ClinicalTrials.gov ↗Join the Salla disease community →

Specialists

1 foundView all specialists →
AM
Adeline Vanderver, MD
Los Angeles, California
Specialist

Rare Disease Specialist

PI on 4 active trials1 Salla disease publication

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 Salla disease.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Salla disease

No recent news articles for Salla 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 is the expected progression of symptoms for my child's specific case of Salla disease?,What therapies (physical, occupational, speech) should we start, and how often?,Are there any clinical trials or research studies we could participate in?,How should we monitor for and manage seizures?,What educational and support services are available for my child?,Should other family members be tested to see if they are carriers?,What should we do in case of a medical emergency related to this condition?

Common questions about Salla disease

What is Salla disease?

Salla disease, also known as free sialic acid storage disease or sialuria Finnish type, is a rare inherited metabolic disorder that belongs to the group of lysosomal storage diseases. It is caused by a problem with how the body handles a sugar molecule called sialic acid (also known as N-acetylneuraminic acid). Normally, sialic acid is transported out of tiny compartments in cells called lysosomes after it has been used. In Salla disease, this transport system does not work properly, so sialic acid builds up inside the lysosomes, especially in the brain and other organs. This buildup damages c

How is Salla disease inherited?

Salla disease follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Salla disease typically begin?

Typical onset of Salla disease is infantile. Age of onset can vary across affected individuals.

Which specialists treat Salla disease?

1 specialists and care centers treating Salla disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.