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Phase 3 Study of Danicopan, an Oral Complement Factor D Inhibitor, As Add-on Therapy to a C5 Inhibitor in Patients with Paroxysmal Nocturnal Hemoglobinuria with Clinically Evident Extravascular Hemolysis

Austin G Kulasekararaj - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Paroxysmal nocturnal hemoglobinuria

N/A

Incomplete Complement Inhibition in Patients with PNH on Eculizumab - 5 Year Experience from the National PNH Service Leeds

Rachael Jones - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

N/A

Eculizumab

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

Alexander Rth - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Paroxysmal nocturnal hemoglobinuria

Eculizumab

Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia in the Phase 3 Hgb-207 Study

John B. Porter - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Transfusion-dependent β-thalassemia

Plerixafor

Validation of a Predictive Score of Acute Chest Syndrome (presev-2 study) in Adults

Christian Kassasseya - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Cardiovascular (CVS)

Acute chest syndrome

Morphine

Higher Hemoglobin Levels Achieved with Voxelotor Are Associated with Lower Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE Study

Elliott Vichinsky - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

N/A

Hydroxyurea

An Age Analysis of the International Sickle Cell World Assessment Survey (SWAY), Focusing on Symptoms, Treatment Goals and the Impact of SCD on Patients’ Daily Lives

Raffaella Colombatti - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

N/A

Improvement in the Clinical Global Impression of Change with Voxelotor in Patients with Sickle Cell Disease in the Phase 3 HOPE Trial

Wally R Smith - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

N/A

Symptoms and Complications of Sickle Cell Disease Analyzed By Patient-Reported Genotype, Sex and Country/Region: Results from the International Sickle Cell World Assessment Survey (SWAY)

Ifeyinwa Osunkwo - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

N/A

Effectiveness of Comprehensive Newborn Screening Program of Sickle Cell Disease on the Childhood Morbidity and Mortality of the Disease: A Systematic Review and Meta-Analysis

Almuhalb Ismail - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Central Nervous System (CNS)

Sickle cell anemia

N/A

Comprehensive Description of Monoallelic GP1BA Variants Associated with Thrombocytopenia

Rachel C Peck - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Central Nervous System (CNS)

Bernard-Soulier syndrome

N/A

A Phase II Study to Assess the Sustained Response Off Treatment in Patients with ITP Receiving Eltrombopag, Who Had a Previous Insufficient Response to Corticosteroids (TAPER): A Recruitment Update

Nichola Cooper - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Infectious Diseases (ID)

Immune thrombocytopenia

Eltrombopag

Outcomes of Patients with Worsening Acquired Thrombotic Thrombocytopenic Purpura Despite Daily Therapeutic Plasma Exchange in the Phase 3 Hercules Trial

Marie Scully - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Musculoskeletal

Thrombotic thrombocytopenic purpura

ALX-0081

Patients’ Reported Perceptions on Satisfaction with Immune Thrombocytopenia Treatments: Results from the ITP World Impact Survey (I-WISh)

Waleed Ghanima - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Autoimmune (AI)

Immune thrombocytopenia

N/A

Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design

Barbara A. Konkle - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

rFVIII

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Amy D Shapiro - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

SIG-001

Real-World Clinical and Laboratory Characteristics in Severe Hemophilia a Patients Commencing Emicizumab

Gavin Ling - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

FVIII

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Sylvia Von Mackensen - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophilia

N/A

Efficacy and Safety of Dabigatran in the Treatment and Secondary Prophylaxis of Children with Venous Thromboembolism and Thrombophilia

Leonardo R. Brandao - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Cardiovascular (CVS)

Venous thrombosis

Heparin

Pharmacokinetic Modelling and Simulation Support the Age and Body-Weight-Adjusted Dosing of Dabigatran Etexilate in Pediatric Patients with Venous Thromboembolism

Daniel Rshammar - American Society of Hematology (ASH) , 2020

Therapeutic Area

Disease

Drug/Treatment

Cardiovascular (CVS)

Venous thrombosis

Pradax

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