Pharming Group (Netherlands)

Recent papers from Pharming Group (Netherlands)

1. Sirolimus and Immunoglobulin Replacement Therapy in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS): A Systematic Literature ReviewAdvances in Therapy · 2026
2. CRISPR activation reveals SOX5/6/9 as key transcriptional regulators directing iPSC-derived cells toward a notochordal lineageStem Cell Reports · 2026
3. Diverse Trial Designs, Populations, and Outcomes: A Systematic Literature Review of Trials for the Treatment of Hereditary Angioedema AttacksAdvances in Therapy · 2026
4. Leniolisib, a Selective Phosphoinositide 3-Kinase Delta (PI3Kδ) Inhibitor, in Japanese Patients Aged 12 and Over with Activated PI3Kδ Syndrome (APDS): Data from a 12-Week, Open-Label, Single-Arm StudyJournal of Human Immunity · 2026
5. Associations between disease factors and clinical outcomes in common variable immunodeficiency: a systematic literature reviewImmunological Medicine · 2026
6. Post Hoc Analysis of Recombinant C1 Inhibitor Clinical Data Using Contemporary Endpoints for Hereditary AngioedemaAdvances in Therapy · 2026
7. Evaluating the impact of leniolisib treatment on symptoms and health-related quality of life in activated phosphoinositide 3-kinase delta (PI3Kδ) syndromeFrontiers in Immunology · 2026
8. Comparative Efficacy of Recombinant C1 Inhibitor Versus Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks: A Matching-Adjusted Indirect Treatment Comparison.2026
9. Pharmacist-Led Discharge Care to Reduce Postdischarge Health Care UtilizationJAMA Network Open · 2026
10. Diagnostic odyssey of patients with the rare immunodeficiency activated PI3 kinase delta syndrome (APDS): case study from expert and patient surveysFrontiers in Immunology · 2026
11. Patient-reported disease burden and health care utilization of HAE-nl-C1INH: insights from a real-world surveyClinical and Experimental Medicine · 2026
12. Associations Between Short‐Term Changes in Disease Activity and Long‐Term Clinical Outcomes in Activated Phosphoinositide 3‐Kinase Delta SyndromeJournal of Immunology Research · 2026
13. Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndromeJournal of Human Immunity · 2025
14. Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndromeJournal of Human Immunity · 2025
15. Natural history of clinical manifestations in activated phosphoinositide 3-kinase δ syndrome (APDS): Time-to-event analyses using the European Society for Immunodeficiencies-APDS registryClinical Immunology · 2025
16. Clinical and genetic findings of individuals tested using the navigateAPDS genetic testing programAnnals of Allergy Asthma & Immunology · 2025
17. Systematic literature reviews to identify epidemiological, clinical, economic and health-related quality of life evidence in activated PI3Kδ syndrome (APDS)BMC Immunology · 2025
18. Development and validation of a predictive 6-min walk score in patients with idiopathic pulmonary fibrosisEuropean Respiratory Journal · 2025
19. The Impact of Activated Phosphoinositide 3-Kinase δ Syndrome (APDS) on Health-Related Quality of Life (HRQoL): Elicitation of Health State Utility Values Through Time Trade-Off (TTO) and EQ-5DAdvances in Therapy · 2025
20. Phosphoinositide-3-kinase δ as an immune check-pathway in cancer Immunology. Therapeutic prospectsExpert Review of Clinical Immunology · 2025