OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:352497
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

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

Overview

Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency is an extremely rare genetic brain disorder that affects movement and thinking abilities. This condition is now classified under broader categories of DNAJC6-related neurodegeneration, which is why it carries an 'obsolete' label in some databases, but the underlying disease is still very real for affected individuals. The DNAJC6 gene provides instructions for making a protein called auxilin, which plays an important role in how brain cells communicate with each other. When this gene does not work properly, it leads to the gradual loss of nerve cells that produce dopamine — a chemical messenger essential for smooth, controlled movement. This is similar to what happens in Parkinson's disease, but it begins much earlier in life, typically during childhood or the teenage years. Key symptoms include progressive stiffness and slowness of movement (parkinsonism), tremor, difficulty walking, intellectual disability, and sometimes seizures. The disease tends to get worse over time. Treatment is mainly supportive, as there is currently no cure. Some patients may respond partially to levodopa (a medication commonly used in Parkinson's disease), but the response is often limited or decreases over time. Physical therapy, speech therapy, and educational support are important parts of managing daily life.

Key symptoms:

Slowness of movementMuscle stiffness and rigidityTremorDifficulty walking and balance problemsIntellectual disabilitySeizures or epilepsySpeech difficultiesDifficulty swallowingLoss of previously learned skillsAbnormal posture (dystonia)Behavioral changesPoor coordinationReduced facial expression

Inheritance

Autosomal recessive

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

Age of Onset

Juvenile

Begins in the teen years

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency.

View clinical trials →

No actively recruiting trials found for OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency at this time.

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

Search ClinicalTrials.gov ↗Join the OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency community →

No specialists are currently listed for OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency.

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 OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency.

Search all travel grants →NORD Financial Assistance ↗

Community

Open OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiencyForum →

No community posts yet. Be the first to share your experience with OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency.

Start the conversation →

Latest news about OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency

No recent news articles for OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency.

Follow this condition to be notified when news becomes available.

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.How quickly do you expect my child's symptoms to progress?,Would a trial of levodopa or other Parkinson's medications be appropriate?,What therapies (physical, speech, occupational) should we start right away?,Are there any clinical trials or research studies my child could participate in?,How should we manage seizures if they develop?,What signs should prompt us to seek emergency care?,Can you refer us to a movement disorder specialist experienced with childhood parkinsonism?

Common questions about OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency

What is OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency?

Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency is an extremely rare genetic brain disorder that affects movement and thinking abilities. This condition is now classified under broader categories of DNAJC6-related neurodegeneration, which is why it carries an 'obsolete' label in some databases, but the underlying disease is still very real for affected individuals. The DNAJC6 gene provides instructions for making a protein called auxilin, which plays an important role in how brain cells communicate with each other. When this gene does not work properly, it leads to

How is OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency inherited?

OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency typically begin?

Typical onset of OBSOLETE: Juvenile parkinsonism with intellectual disability due to DNAJC6 deficiency is juvenile. Age of onset can vary across affected individuals.