Sarlósejtes vérszegénység: ki dolgozik rajta

Intézményenként egy kártya. Célpontra vagy országra szűkítheted a listát.

• Minden célpont
• G6PD 0
• NLRP3 0
• APOL1 0
• CD19 3
• BTK 0
• FGFR 0
• NRF2 0
• SGLT2 1
• CFTR 1
• CXCR4 0
• DMD 1
• GLP-1 0
• HDAC 1
• HMGB1 0
• HTT 1
• JAK 0
• NF1 1
• SMN1 1

726 intézményből 24, a friss publikációk és a nyitott vizsgálatok szerint rendezve. Ez nem minőségi rangsor.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• European Commission (Horizon Europe) · HORIZON-ERC

  Innovating Sickle Cell Disease Treatment: From Non-Canonical Signaling and Immune Activation to Personalized Targeted Therapies (TARGET-SCD)

  Universite Paris Cite· Paris, Franciaország· EU contribution 2 449 320 EUR· until 2031. dec.

• European Commission (Horizon Europe) · HORIZON-TMA-MSCA-PF-EF

  Deciphering Inflammatory and Placental Mechanisms of Pre-eclampsia in Sickle Cell Disease (PRECISE)

  Imagine Institut des Maladies Genetiques Necker Enfants Malades Fondation· Paris, Franciaország· EU contribution 242 261 EUR· until 2028. dec.· 2 countries

• European Commission (Horizon Europe) · HORIZON-ERC

  A metabolic-based defense strategy against malaria (KILLBILL)

  Fundacao Gimm - Gulbenkian Institute for Molecular Medicine· Lisboa, Portugália· EU contribution 2 500 000 EUR· until 2031. szept.

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-JU-CSA

  CONSORTIUM FOR DEVELOPING REGULATORY CAPACITY FOR CLINICAL TRIALS USING GENE THERAPY PRODUCTS AND STRENGTHENING PHARMACOVIGILANCE IN THE CONDUCT OF CLINICAL TRIALS IN EAST AFRICA (CAPACITY 2023)

  Universidade de Coimbra· Coimbra, Portugália· EU contribution 999 315 EUR· until 2027. márc.· 3 countries

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-RIA

  Synthetic generation of hematological data over federated computing frameworks (SYNTHEMA)

  Universidad Politecnica de Madrid· Madrid, Spanyolország· EU contribution 6 514 560 EUR· until 2026. nov.· 10 countries

  The disease is named only in the funder's project summary, not in the title.

5 of 8 running projects, newest first. Listed amounts together: 25,5 M EUR.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Leállított, visszavont és felfüggesztett vizsgálatok, a szponzor által a regiszterben megadott okkal, szó szerint idézve.

• Felfüggesztve

  Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease

  NCT07436767· Institute of Hematology & Blood Diseases Hospital, China· Frissítve: 2026. nov. 5.

  “Difficulty identifying eligible patients.”A regiszter szövege, változtatás nélkül. Forrás

• Leállították

  Open-Label Extension Study to Pioneer Study 6058-SCD-101

  NCT07401823· Fulcrum Therapeutics· Frissítve: 2026. okt. 28.

  “Sponsor Decision”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

  NCT03279094· City of Hope Medical Center· Frissítve: 2026. okt. 26.

  “Closed to accrual - no accruals in CTMS”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease

  NCT06979492· Emory University· Frissítve: 2026. okt. 19.

  “The study was withdrawn due to no enrollment”A regiszter szövege, változtatás nélkül. Forrás

• Leállították

  AB1 in Adult Patients With Sickle Cell Disease (SCD)

  NCT05261711· Nirmish Shah· Frissítve: 2026. okt. 15.

  “Study is terminated prior to its planned completion as anticipated by the protocol due to enrollment completion. Data clean up and analysis is being performed.”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Biotin-Acridine Red Cell Exchange Kinetics

  NCT07155031· Cerus Corporation· Frissítve: 2026. szept. 22.

  “This study has been suspended due to a lack of funding. The protocol will be reconsidered within the next 6-12 months if/as funding becomes available.”A regiszter szövege, változtatás nélkül. Forrás

• Visszavonták

  Testing the Sickle Cell Caregiver Collaboration for Child Development (SCCCD) Intervention

  NCT06562439· Washington University School of Medicine· Frissítve: 2026. aug. 25.

  “not a clinical trial”A regiszter szövege, változtatás nélkül. Forrás

• Leállították

  Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

  NCT07431398· Fulcrum Therapeutics· Frissítve: 2026. jún. 18.

  “Sponsor decision”A regiszter szövege, változtatás nélkül. Forrás

• Leállították

  Siplizumab for Sickle Cell Disease Transplant

  NCT06078696· Columbia University· Frissítve: 2026. jún. 10.

  “Closed by the industry collaborator”A regiszter szövege, változtatás nélkül. Forrás

• Leállították

  GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)

  NCT05632354· Pfizer· Frissítve: 2026. márc. 19.

  “Study terminated as data obtained from this non-placebo-controlled trial may not be informative for the interpretation of the benefits and risks of osivelotor”A regiszter szövege, változtatás nélkül. Forrás

Vizsgálatok, amelyek regiszteradata szerint az egyéni résztvevői adatokat (IPD) megosztják.

• Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease

  NCT07616154· St. Jude Children's Research Hospital· Forrás

• Efficacy of Liberal Versus Restricted IV Fluid Approach in the Management of Sickle Cell Vaso-Occlusive Crisis

  NCT07188766· Imam Abdulrahman Bin Faisal University· Forrás

• Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE)

  NCT04084080· University of Pittsburgh· Forrás

• A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)

  NCT06439082· Novartis Pharmaceuticals· Forrás

• Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease

  NCT06665997· University of Pennsylvania· Forrás

• Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)

  NCT07224360· CSL Behring· Forrás

• A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

  NCT06546670· Novartis Pharmaceuticals· Forrás

• The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

  NCT06975865· Sanofi· Forrás

• Gene Editing For Sickle Cell Disease

  NCT06506461· St. Jude Children's Research Hospital· Forrás

• Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

  NCT03814746· Novartis Pharmaceuticals· Forrás

Publikációk erről a betegségről egy MeSH-módszerkifejezés szerint, például szekvenálási vagy képalkotó módszerre.

• Single-Cell Analysis 1
• CRISPR-Cas Systems 15
• Gene Expression Profiling 7
• Exome Sequencing 1
• Proteomics 7
• Metabolomics 2
• Mass Spectrometry 2
• Flow Cytometry 5
• Immunohistochemistry 2
• Magnetic Resonance Imaging 21
• Machine Learning 5
• Deep Learning 3
• Cohort Studies 19
• Genome-Wide Association Study 5

• Changes in circular RNA expression in acute chest syndrome and vaso-occlusive crisis in sickle cell disease: analysis of a public RNA-seq cohort.

  Expert Rev Hematol, 2026. máj.

• Exploring plasma microRNA profiling and signaling pathways in comorbidity-free sickle cell anemia: a pilot case-control study.

  BMC Res Notes, 2026. márc.

• Gene expression profiling identifies potential biomarkers for vaso-occlusive episodes in sickle cell disease.

  JCI Insight, 2026. márc.

• Exploring erythroid cell transcriptomics to understand regulation of fetal hemoglobin expression for advanced sickle cell disease treatment.

  Hum Mol Genet, 2026. febr.

• Whole Blood Transcriptomic Analysis of Sickle Cell Trait.

  Eur J Haematol, 2026. jan.

• The induction effect of hydroxyurea and metformin on fetal globin in the K562 cell line.

  Mol Med, 2025. ápr.

• Metformin induces FOXO3-dependent fetal hemoglobin production in human primary erythroid cells.

  Blood, 2018. jún.

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