Inborn errors of immunity: Manifestation, treatment, and outcome—an ESID registry 1994–2024 report on 30,628 patients
Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial
Current genetic diagnostics in inborn errors of immunity
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
Diagnostic evaluation of paediatric autoimmune lymphoproliferative immunodeficiencies (ALPID): a prospective cohort study
A patient empowerment program for primary immunodeficiency improves quality of life in children and adolescents
Gene Therapy in Patients with the Crigler–Najjar Syndrome
Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry and comparison with other autoimmune-lymphoproliferative inborn errors of immunity
Prospective Newborn Screening for SCID in Germany: A First Analysis by the Pediatric Immunology Working Group (API)
Liver Transplantation for Primary Sclerosing Cholangitis (PSC) With or Without Inflammatory Bowel Disease (IBD)—A European Society of Organ Transplantation (ESOT) Consensus Statement
Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis
Noninvasive scores are poorly predictive of histological fibrosis in paediatric fatty liver disease
Genetic landscape of pediatric acute liver failure of indeterminate origin
LMS-Based Pediatric Reference Values for Parameters of Phosphate Homeostasis in the HARP Cohort
The GAIN Registry — a New Prospective Study for Patients with Multi-organ Autoimmunity and Autoinflammation
Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial
Prognosis of Children Undergoing Liver Transplantation: A 30-Year European Study
Human CARMIL2 deficiency underlies a broader immunological and clinical phenotype than CD28 deficiency
Case Report: Rubella Virus-Induced Cutaneous Granulomas in Two Pediatric Patients With DNA Double Strand Breakage Repair Disorders – Outcome After Hematopoietic Stem Cell Transplantation
Defining paediatric metabolic (dysfunction)-associated fatty liver disease: an international expert consensus statement
Diagnosis and management of secondary causes of steatohepatitis
Multisystem inflammation and susceptibility to viral infections in human ZNFX1 deficiency
European Liver Transplant Registry: Donor and transplant surgery aspects of 16,641 liver transplantations in children
Effects of odevixibat on pruritus and bile acids in children with cholestatic liver disease: Phase 2 study
Current Practices on Diagnosis, Prevention and Treatment of Post-Transplant Lymphoproliferative Disorder in Pediatric Patients after Solid Organ Transplantation: Results of ERN TransplantChild Healthcare Working Group Survey
COVID-19 related reduction in pediatric emergency healthcare utilization – a concerning trend
rs641738C>T near MBOAT7 is associated with liver fat, ALT and fibrosis in NAFLD: A meta-analysis
Impact of COVID‐19 on liver transplantation in Europe: alert from an early survey of European Liver and Intestine Transplantation Association and European Liver Transplant Registry
Pediatric transplantation in Europe during the COVID‐19 pandemic: Early impact on activity and healthcare