Progressive supranuclear palsy-progressive non-fluent aphasia syndrome

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ORPHA:240112OMIM:260540G23.1
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4Specialists8Treatment centers

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Overview

Progressive supranuclear palsy-progressive non-fluent aphasia syndrome (PSP-PNFA) is a rare brain disorder that combines features of two conditions: progressive supranuclear palsy (PSP) and progressive non-fluent aphasia (PNFA). PSP is a condition that affects movement, balance, and eye movements, while PNFA is a type of language disorder where a person gradually loses the ability to speak fluently. In PSP-PNFA, patients typically develop increasing difficulty producing speech — words come out slowly, with effort, and sentences may be grammatically incorrect or broken. Over time, patients may also develop problems with balance, stiffness in the body, difficulty looking up or down (called vertical gaze palsy), and trouble swallowing. The disease is caused by an abnormal buildup of a protein called tau in certain areas of the brain, which damages nerve cells over time. This condition is part of a group of diseases known as tauopathies. There is currently no cure for PSP-PNFA. Treatment focuses on managing symptoms, such as speech therapy to help maintain communication as long as possible, physical therapy for balance and mobility issues, and medications to help with stiffness or mood changes. The disease tends to progress over several years, and a team of specialists is usually needed to provide the best care.

Also known as:

Key symptoms:

Slow, effortful speechDifficulty forming grammatically correct sentencesProblems finding the right wordsDifficulty moving the eyes, especially looking up or downBalance problems and frequent fallsStiffness in the neck, trunk, or limbsTrouble swallowingChanges in personality or behaviorSlowed movementsDifficulty with fine motor tasks like writingFacial stiffness or reduced facial expressionDepression or apathyCognitive decline over time

Clinical phenotype terms (21)— hover any for plain English
Speech apraxiaHP:0011098Deficit in grammarHP:0006977Spoken word recognition deficitHP:0030391Deficit in phonologic short-term memoryHP:0002549Abnormal prosodyHP:0031434Progressive extrapyramidal muscular rigidityHP:0007158Vertical supranuclear gaze palsyHP:0000511Anomic aphasiaHP:0030784StutteringHP:0025268Limb apraxiaHP:0030217
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Late onset

Begins later in life, typically after age 50

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Progressive supranuclear palsy-progressive non-fluent aphasia syndrome.

View clinical trials →

No actively recruiting trials found for Progressive supranuclear palsy-progressive non-fluent aphasia syndrome at this time.

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Specialists

4 foundView all specialists →
AP
Adam L Boxer, MD, PhD
SAN FRANCISCO, CA
Specialist
PI on 2 active trials
AP
Adam L. Boxer, M.D., Ph.D.
SAN FRANCISCO, CA
Specialist
PI on 2 active trials

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 supranuclear palsy-progressive non-fluent aphasia syndrome.

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Community

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Latest news about Progressive supranuclear palsy-progressive non-fluent aphasia syndrome

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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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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What stage of the disease am I in, and what changes should I expect over the next year or two?,What therapies — such as speech therapy and physical therapy — should I start now to maintain my abilities as long as possible?,Are there any clinical trials for PSP or tauopathies that I might be eligible for?,What medications might help with my stiffness, balance, or mood symptoms?,When should we start planning for communication aids or assistive devices?,What steps should we take now for advance care planning?,What resources or support groups are available for me and my family?

Common questions about Progressive supranuclear palsy-progressive non-fluent aphasia syndrome

What is Progressive supranuclear palsy-progressive non-fluent aphasia syndrome?

Progressive supranuclear palsy-progressive non-fluent aphasia syndrome (PSP-PNFA) is a rare brain disorder that combines features of two conditions: progressive supranuclear palsy (PSP) and progressive non-fluent aphasia (PNFA). PSP is a condition that affects movement, balance, and eye movements, while PNFA is a type of language disorder where a person gradually loses the ability to speak fluently. In PSP-PNFA, patients typically develop increasing difficulty producing speech — words come out slowly, with effort, and sentences may be grammatically incorrect or broken. Over time, patients may

How is Progressive supranuclear palsy-progressive non-fluent aphasia syndrome inherited?

Progressive supranuclear palsy-progressive non-fluent aphasia syndrome follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Progressive supranuclear palsy-progressive non-fluent aphasia syndrome typically begin?

Typical onset of Progressive supranuclear palsy-progressive non-fluent aphasia syndrome is late onset. Age of onset can vary across affected individuals.

Which specialists treat Progressive supranuclear palsy-progressive non-fluent aphasia syndrome?

4 specialists and care centers treating Progressive supranuclear palsy-progressive non-fluent aphasia syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.