Latest Research in Hemoglobinopathies and Related Disorders

25 research papers · 2026 median publication year

Top Research Topics in Hemoglobinopathies and Related Disorders

Highest-Cited Papers

  1. CRISPR-Based Mediated Reactivation of Fetal Hemoglobin as a Therapeutic Strategy for Hemoglobinopathies: Evidence from Preclinical to Clinical Trials in Sickle Cell Disease and β-Thalassemia
  2. Zebras in Plain Sight: Rare Disease Knowledge and Awareness Among Pediatric and Family Medicine Resident Physicians
  3. Rare diseases in Brazil: a nationwide analysis of the diagnostic odyssey
  4. Beyond the Prevention of Anti-drug Antibody Formation with Uricase Therapy: Mechanistic Roles of DMARDs in Modifying Gout Flare Risk
  5. Beyond Primary Epilepsy: Fahr's Syndrome Secondary to Congenital Parathyroid Agenesis Presenting With Generalised Seizures in a Young Adult
  6. Normal cholestanol in a genetically confirmed cerebrotendious xanthomatosis case presenting as neonatal jaundice
  7. Hyperkinetic Movements in an Infant with Citrullinemia Type 1
  8. Neutrophil Fate and Function in Gout: From Sterile Inflammation to Resolution
  9. Mitochondrial Damage in Hemoglobinopathies: The Role of Cell-Free mtDNA as a Potential Biomarker
  10. Sex Difference in the Impact of Steatotic Liver Disease on Incident Hepatocellular Carcinoma in Patients with Chronic Hepatitis B
  11. Precision Medicine in Pediatric Nephrology: From Shared Clinical Phenotypes to Genotype-Guided Diagnosis and Management
  12. Plasma growth differentiation factor 15 levels are more strongly associated with host physiological status than with tumor progression in colorectal cancer
  13. Preclinical evaluation of entecavir as a fetal hemoglobin inducer using in vitro, in vivo, network pharmacology, and molecular docking approaches
  14. Digenic disease causing variants in a patient with severe nephrocalcinosis
  15. Renal and Systemic Complications in Autosomal Dominant Hypocalcaemia Type 1: A Case Series from North East England
  16. Sitosterolemia in pregnancy: A rare lipid disorder and its obstetric management
  17. A case report of WT1-associated infantile steroid-resistant nephrotic syndrome with atypical genotype–phenotype correlation
  18. UMOD gene variants in autosomal dominant tubulointerstitial kidney disease: first cases reported from Türkiye
  19. Genome-wide association study of restless legs syndrome in end-stage renal disease
  20. Beyond cirrhosis: Major adverse liver outcomes across homozygous and heterozygous Alpha-1 antitrypsin deficiency associated liver disease
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L3 Region - - 2026 Sep Q3

Hemoglobinopathies and Related Disorders

25 papers

Top Topics (10)

Hemoglobinopathies and Related Disorders3
Genomics and Rare Diseases2
Gout, Hyperuricemia, Uric Acid2
Thyroid and Parathyroid Surgery2
Cholesterol and Lipid Metabolism2
Kidney Stones and Urolithiasis Treatments2
Parathyroid Disorders and Treatments2
Metabolism and Genetic Disorders1
Mitochondrial Function and Pathology1
Liver Disease Diagnosis and Treatment1

Top Publications (20)

1.CRISPR-Based Mediated Reactivation of Fetal Hemoglobin as a Therapeutic Strategy for Hemoglobinopathies: Evidence from Preclinical to Clinical Trials in Sickle Cell Disease and β-Thalassemia2.Zebras in Plain Sight: Rare Disease Knowledge and Awareness Among Pediatric and Family Medicine Resident Physicians3.Rare diseases in Brazil: a nationwide analysis of the diagnostic odyssey4.Beyond the Prevention of Anti-drug Antibody Formation with Uricase Therapy: Mechanistic Roles of DMARDs in Modifying Gout Flare Risk5.Beyond Primary Epilepsy: Fahr's Syndrome Secondary to Congenital Parathyroid Agenesis Presenting With Generalised Seizures in a Young Adult6.Normal cholestanol in a genetically confirmed cerebrotendious xanthomatosis case presenting as neonatal jaundice7.Hyperkinetic Movements in an Infant with Citrullinemia Type 18.Neutrophil Fate and Function in Gout: From Sterile Inflammation to Resolution9.Mitochondrial Damage in Hemoglobinopathies: The Role of Cell-Free mtDNA as a Potential Biomarker10.Sex Difference in the Impact of Steatotic Liver Disease on Incident Hepatocellular Carcinoma in Patients with Chronic Hepatitis B11.Precision Medicine in Pediatric Nephrology: From Shared Clinical Phenotypes to Genotype-Guided Diagnosis and Management12.Plasma growth differentiation factor 15 levels are more strongly associated with host physiological status than with tumor progression in colorectal cancer13.Preclinical evaluation of entecavir as a fetal hemoglobin inducer using in vitro, in vivo, network pharmacology, and molecular docking approaches14.Digenic disease causing variants in a patient with severe nephrocalcinosis15.Renal and Systemic Complications in Autosomal Dominant Hypocalcaemia Type 1: A Case Series from North East England16.Sitosterolemia in pregnancy: A rare lipid disorder and its obstetric management17.A case report of WT1-associated infantile steroid-resistant nephrotic syndrome with atypical genotype–phenotype correlation18.UMOD gene variants in autosomal dominant tubulointerstitial kidney disease: first cases reported from Türkiye19.Genome-wide association study of restless legs syndrome in end-stage renal disease20.Beyond cirrhosis: Major adverse liver outcomes across homozygous and heterozygous Alpha-1 antitrypsin deficiency associated liver disease
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