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Rare and hereditary disorders
Primary immunodeficiencies (severe)
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Publications and studies (2085)
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema (2026-05-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Observational Study of Subcutaneous Immunoglobulin (Cutaquig) in Patients With Primary and Secondary Immunodeficiency. (2026-05-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema (2026-05-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Project Khanya: Peer-Delivered Intervention to Improve HIV Medication Adherence and Substance Use in South Africa (2026-05-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE (2026-05-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Identification of New Inborn Errors of Immunity (2026-05-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Pathophysiology of Inborn Immunodeficiencies (2026-05-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Open-Label Extension of EryDex Study IEDAT-04-2022 (2026-05-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients (2026-05-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity (2026-05-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Usage of Spirometry in Managing IgG Therapy in CVID With Airway Disease (2026-05-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluate the Neurological Effects of EryDex on Subjects With A-T (2026-04-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. HAE Burden and Crisis Management (2026-04-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Prevalence, Incidence and Risk Signature of Chronic Kidney Disease in Sub-Saharan Africa (2026-04-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector (2026-04-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Systematic Search for Primary Immunodeficiency in Adults With Infections (2026-04-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Improving the Diagnosis of Common Variable Immune Deficiency (2026-04-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Phase 2 in Adult Subjects With Hereditary Angioedema (2026-04-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs) (2026-04-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT (2026-04-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal Prognosis of Lymphoproliferative Manifestations in Primary Immunodeficiencies (2026-04-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases (2026-04-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Immunoglobulin Efficacy and Immune Profiling in Antibody Immunodeficiency (2026-04-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Trial Comparing Two Strategies of Vaccination Against Hepatitis B in HIV-infected Patients Non Responding to Primary Immunization (B-BOOST) (2026-04-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. National Multicenter Trial Evaluating Two Treatments in Patients With Primary Human Immunodeficiency Virus (HIV-1) Infection (2026-04-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics (2026-04-01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland (2026-03-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study (2026-03-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) (2026-03-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (2026-03-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. NTLA-2002 in Adults With Hereditary Angioedema (HAE) (2026-03-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation (2026-03-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Targeting Real World Usage In Stroke Treatment (2026-03-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gene Therapy for X-linked Severe Combined Immunodeficiency (2026-03-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Primary Immunodeficiency and Pregnancy (2026-03-09) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Real-life Ecological Momentary Assessment of Lived Burden in Hereditary Angioedema (2026-03-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Integrated Health Services (IHS): A Structural Intervention to Improve HIV/STI Screening and PrEP Navigation in Primary Care (2026-03-03) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL) (2026-02-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Berotralstat Treatment in Children With Hereditary Angioedema (2026-02-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T) (2026-02-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. ENHANCE-EvideNce Led Co-created HeAlth Systems interventioNs for MLTCs CarE (2026-02-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Combination Primary Care and Prevention Services for Women Who Inject Drugs and Exchange Sex in Seattle, Washington (2026-02-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Immune Disorder HSCT Protocol (2026-02-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts (2026-02-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Immunoglobulin G Therapy Dose Optimization (2026-02-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biologics And Clinical Immunology Cohort at Sinai (2026-02-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE (2026-02-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Autologous Gene Therapy for Artemis-Deficient SCID (2026-02-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation (2026-02-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Leniolisib for Immune Dysregulation in PIDs (2026-02-13) ♡
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