Clinical Trials for Swedish Orphan Biovitrum

Pacritinib With Standard of Care Azacitidine or Decitabine as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant for Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF

Assessment of Macrophage Activation syndromE in STill's Disease in Italy

A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)

Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A

Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome

A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis

Emapalumab Prevention of CAR-T Cell Associated Toxicities

Prevalence of Synovitis in Patients With Haemophilia A

Avatrombopag vs. Placebo for CIT in GI Malignancies

A Trial Comparing the Efficacy and Safety of Anakinra Versus Intravenous Immunoglobulin (IVIG) Retreatment, in Patients With Kawasaki Disease Who Failed to Respond to Initial Standard IVIG Treatment

A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa

A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD)

ITP Registry and Accompanying Biospecimen Collection

A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health

Effects of Anakinra in Subjects With Corticosteroid-resistant/Intolerant Meniere's Disease and Autoimmune Inner Ear Disease

A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation