3,939 results on '"Hemoglobinuria, Paroxysmal"'
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2. Cancer in Inherited Bone Marrow Failure Syndromes
3. A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment (ACCESS-1)
4. A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
5. Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
6. Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
7. Specified Drug-use Surveillance of Fabhalta Capsules
8. Home Reported Outcomes in PNH
9. Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)
10. Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment
11. Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab
12. Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
13. A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
14. Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
15. BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy (REDEEM-2)
16. Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
17. Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms (CONSENTII)
18. ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
19. Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
20. Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
21. A Real-world, Multi-center, Prospective, Observational Study for PNH in China (ReWoPNH)
22. A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
23. A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition (COMMODORE 3)
24. Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. (APPULSE)
25. A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
26. Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria (COMPOSER)
27. Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients (PNH)
28. A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors (COMMODORE 1)
29. A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. (COMMODORE 2)
30. Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China (Soliris)
31. Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
32. To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
33. Managed Access Programs for LNP023, Iptacopan
34. Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
35. Real-life Treatment Outcomes of Ravulizumab in PNH (PNH-RECORD)
36. Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (APPLY-PNH)
37. Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (APPOINT-PNH)
38. Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
39. Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
40. Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
41. Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
42. Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (ACCESS 2)
43. A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
44. Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
45. Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
46. Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects
47. Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
48. To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects
49. A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (COMPLETE)
50. Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
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