Activated PI3K-delta syndrome 1

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ORPHA:693661OMIM:615513
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1FDA treatments8Treatment centers1Financial resources

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Overview

Also known as:

Senescent T-cells-lymphadenopathy-immunodeficiency syndrome due to p110delta activating mutations, type 1Activated p110delta syndrome, type 1APDS1APDS type 1Activated phosphoinositide 3-kinase delta syndrome-1PASLI-CD

Clinical phenotype terms— hover any for plain English:

Decreased total B cell countHP:0010976Intestinal lymphoid nodular hyperplasiaHP:0011956DacryocystitisHP:0000620Recurrent candida infectionsHP:0005401Recurrent tonsillitisHP:0011110Persistent human papillomavirus infectionHP:0020114Molluscum contagiosumHP:0032163Chronic active Epstein-Barr virus infectionHP:0032204Persistent CMV viremiaHP:0032247
Orphanet ↗OMIM ↗NORD ↗

Treatments

1 available

Joenja

leniolisib· Pharming Technologies B.V.

JOENJA is indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older

View Details →FDA Label ↗Copay Card ↗Patient Assistance ↗

Clinical Trials

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No actively recruiting trials found for Activated PI3K-delta syndrome 1 at this time.

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

Search ClinicalTrials.gov ↗Join the Activated PI3K-delta syndrome 1 community →

Specialists

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No specialists are currently listed for Activated PI3K-delta syndrome 1.

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

Financial Resources

1 resources
Joenja(leniolisib)— Pharming Technologies B.V.

Travel Grants

No travel grants are currently matched to Activated PI3K-delta syndrome 1.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Activated PI3K-delta syndrome 1

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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.