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Rare and hereditary disorders
Primary immunodeficiencies (severe)
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Publications and studies (2086)
- CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells. (2023/01/04) ♡
- The chaperone protein p32 stabilizes HIV-1 Tat and strengthens the p-Tefb/RNAPII/TAR complex promoting HIV transcription elongation. (2023/01/03) ♡
- Expanding the accessibility of harm reduction services in the United States: Measuring the impact of an automated harm reduction dispensing machine. (2023/01/01) ♡
- Nef enhances HIV-1 replication and infectivity independently of SERINC5 in CEM T cells. (2023/01/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Gene-environment interactions in birth defect etiology: Challenges and opportunities. (2023/01/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Disseminated multidrug-resistant tuberculosis and SARS-CoV-2 co-infection in a child with IL-12Rβ1 deficiency. (2023/01/01) ♡
- The demographics of autoimmune hepatitis in human immunodeficiency virus-infected patients: a United States cross-sectional study. (2023/01/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Applications of virus-specific T cell therapies post-BMT. (2023/01/01) ♡
- The spectrum of inborn errors of immunity: a single tertiary center retrospective study in Alborz, Iran. (2023/01/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. The Middle East and North Africa Diagnosis and Management Guidelines for Inborn Errors of Immunity. (2023/01/01) ♡
- Effect of nutritional status on outcomes in children receiving umbilical cord blood stem cell transplantation. (2023/01/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Novel sphingosine-1-phosphate lyase mutation causes multisystemic diseases: case report. (2023/01/01) ♡
- Comparison on treatment outcomes of patients enrolled on anti-retroviral therapy at different levels of the health-care system in a high HIV/AIDS setting. (2023/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Investigation of the Effects of Pulmonary Rehabilitation in Children With Primary Immunodeficiency (2023-12-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Home-based Complex Intervention for Children With Ataxia Telangiectasia (2023-11-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Vorinostat and Combination Chemotherapy With Rituximab in Treating Patients With HIV-related Diffuse Large B-Cell Non-Hodgkin Lymphoma or Other Aggressive B-Cell Lymphomas (2023-11-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Visceral Adiposity Measurement and Observation Study (2023-11-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases (2023-11-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects (2023-10-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Peer-led Social Media Intervention to Prevent HIV Among Young Men Who Have Sex With Men (YMSM) (2023-10-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Living With a Long-Term Condition Study (2023-10-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency (2023-10-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in Argentina (2023-10-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naive Participants With Primary Immunodeficiency (2023-09-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant (2023-09-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study (2023-09-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Influence of Primary HIV-1 Drug Resistance Mutations on Immune Reconstruction in PLWH (2023-09-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (2023-09-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO) (2023-09-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Anti-HIV Medications for People Recently Infected With HIV (2023-09-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations (2023-09-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Feasibility of SBIRT-PN (2023-09-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Exercise Capacity, Physical Activity Level, and Quality of Life in Children With Primary Immunodeficiency (2023-08-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of Subcutaneous Immunoglobulin Product Cutaquig in Terms of Safety and Efficacy in the Treatment of Patients With Primary Immunodeficiencies (2023-08-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Online HIV Prevention for Young Male Couples (2023-07-28) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanded Access for the Prevention of Acute Attacks of 1) Hereditary Angioedema (HAE) in Children and 2) Non-histaminergic Angioedema With Normal C1 Inhibitor (C1-INH) in Teenagers and Adults (2023-07-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Ataxia Telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis (2023-07-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Participation in a Research Registry for Immune Disorders (2023-07-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Investigate Efficacy, Safety, and Pharmacokinetics of BT595 in Subjects With PID (2023-07-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Identifying Efficient Linkage Strategies for Men (2023-07-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluating Bronchodilator Response in Patients With Bronchiectasis (2023-07-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks (2023-06-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Innovative Strategies to Increase ART Initiation and Viral Suppression Among HIV-positive Men in Malawi (2023-06-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema (2023-06-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (2023-06-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema (2023-06-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Long Term Safety Study of BCX7353 in Hereditary Angioedema (2023-06-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanded Access Protocol for Table Eleucel for Patients With Epstein-Barr Virus-Associated Viremia or Malignancies (2023-06-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral Immunodeficiency (2023-06-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Engaging Men Through HIV Self-Testing With Differentiated Care to Improve ART Initiation and Viral Suppression Among Men in Malawi (2023-05-15) ♡
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