Results 141 to 150 of about 12,365 (224)

Clinical predictors of mortality in immune thrombotic thrombocytopenic purpura: A National Inpatient Sample analysis

open access: yes
British Journal of Haematology, EarlyView.
Anand Shah   +4 more
wiley   +1 more source

Feasibility and Safety of Venetoclax for Cytoreduction During Induction Therapy in Newly Diagnosed Acute Promyelocytic Leukemia

open access: yesEuropean Journal of Haematology, EarlyView.
ABSTRACT Objective This study aimed to evaluate the feasibility and safety of venetoclax as a cytoreductive strategy during induction therapy in newly diagnosed acute promyelocytic leukemia (APL), and to provide an exploratory description of its early efficacy.
Yu Wang   +3 more
wiley   +1 more source

Angiogenic Imbalance Defines Multisystem Phenotypes of Preeclampsia: A Phenotype-Oriented Cohort Study. [PDF]

open access: yesClin Pract
Tătaru-Copos A   +9 more
europepmc   +1 more source

Deep intronic ANK1 variants causing pseudo‐exon inclusion in hereditary spherocytosis: Whole‐genome sequencing and functional assessment

open access: yes
British Journal of Haematology, EarlyView.
Victor Marin   +8 more
wiley   +1 more source

Prospective Study of Targeted Busulfan–Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Genetic Rare Diseases

open access: yesEuropean Journal of Haematology, EarlyView.
ABSTRACT Objectives Genetic rare diseases (GRDs), including chronic granulomatous disease, familial hemophagocytic lymphohistiocytosis, and congenital neutropenia, often require hematopoietic stem cell transplantation (HSCT) as the only curative option.
Bo Kyung Kim   +6 more
wiley   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

open access: yesHaemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach   +2 more
wiley   +1 more source

Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community

open access: yesHaemophilia, EarlyView.
ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein   +6 more
wiley   +1 more source

Ruptured Subserosal Pyomyoma Presenting as Peritonitis in the Early Postpartum Period: A Case Report. [PDF]

open access: yesClin Med Insights Case Rep
Kebede RG   +5 more
europepmc   +1 more source

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