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Prognostic Value of Clone Size in Paroxysmal Nocturnal Hemoglobinuria (PNH) for Thrombotic Events in Untreated Patients in the International PNH Registry

Regis Peffault De Latour - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Paroxysmal nocturnal hemoglobinuria

N/A

A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria

Robert A. Brodsky - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Paroxysmal nocturnal hemoglobinuria

Eculizumab

Efficacy of Eculizumab in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria in the International PNH Registry

lvaro Urbano-Ispizua - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Paroxysmal nocturnal hemoglobinuria

Eculizumab

RBC-Specific CD47 Pruning Confers Protection and Underlies the Transient Anemia in Patients Treated with Anti-CD47 Antibody 5F9

James Y Chen - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Oncology (ONC)

Acute myelocytic leukemia

N/A

A Review of 1000 Molecular Investigations of Rare Inherited Anaemia and Related Conditions with the Red Cell Gene Panel

Rachel Mayhew - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Hemophagocytic lymphohistiocytosis

N/A

A Fully Human Anti-BMP6 Antibody Reduces the Need for Erythropoietin Stimulating Agent in Two Rodent Anemia of Chronic Disease Models

Verena Petzer - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Nephrology

Chronic kidney disease

Sodium ferric gluconate complex

Effect of Ljpc-401 (synthetic human hepcidin) on Iron Parameters in Healthy Adults

John Porter - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

N/A

LJPC 401

Real-World Experience of Switching from Deferasirox Dispersible to Film-Coated Tablets: Impact on Adherence to Chelation Therapy, Iron Overload and Renal Function

Simon Cheesman - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Endocrine/metabolic Diseases (ME)

Hemochromatosis

Deferasirox

Evaluation of Endocrine Complications in Beta-Thalassemia Intermedia Patients: A Cross Sectional Multi-Center Study

Mehran Karimi - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Ophthalmology (OP)

Beta-thalassemia

Hydroxyurea

Expression of the Human Alpha-Globin Cluster in the Absence of the Major Regulatory Element Mcs-R2

Mohsin Badat - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Dyspnea

N/A

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Maciej W Garbowski - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Thalassemia

Sotatercept

Dose Selection Process for Younger Children Participating in a Phase 3 Study to Evaluate the Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Patients with Sickle Cell Disease

Brinda Tammara - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

rivipansel

Proteomics Pathways of Sickle Cell Anemia (P2SCA): A Comprehensive Analysis By Liquid Chromatography Mass Spectrometry of Erythrocyte Membrane Proteins Characterized from the Muhimbili Sickle Cell Programme, Tanzania

Haddy KS Fye - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

Hydroxyurea

Hemoglobin S Induces Exposure of Red Blood Cell Membrane Skeleton Microdomains Bearing Mannose That Stimulate Phagocytosis By Macrophages: A Molecular Basis for Hemolysis in Sickle Cell Disease but Protection Against Plasmodium Falciparum malaria

Huan Cao - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

N/A

Cerebral Infarcts and Cerebrovascular Disease in Neurologically Intact Tanzanian Children with Sickle Cell Anaemia

Mboka Mwakatika Mwakatika - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Central Nervous System (CNS)

Sickle cell anemia

N/A

Hydroxyurea Therapy Does Not Impact Current Survival Estimates in the East London Sickle Cell Newborn Cohort Study

Paul Telfer - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

N/A

Hydroxyurea

Pregnancy Outcomes in Patients with Sickle Cell Syndromes Managed Antenatally with Exchange Transfusions: A Single Centre Analysis

Evangelia Vlachodimtropoulou Koumoutsea - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Obstetrics/Gynecology

Spontaneous abortion

Allopurinol

Lung Function Impairment in Pediatric Subjects with Sickle Cell Anemia from Nigeria Is Associatede with Associated Low Steady Haemoglobin

Baba PD Inusa - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Acute chest syndrome

N/A

Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey

Giovanna Graziadei - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Gastroenterology (GU)

Gastric antral vascular ectasia

N/A

Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials

Pramod K. Mistry - American Society of Hematology (ASH) , 2018

Therapeutic Area

Disease

Drug/Treatment

Endocrine/metabolic Diseases (ME)

Gaucher disease

Eliglustat

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