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BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.
Eur J Haematol
December 2024
Sheba Medical Center, The Israeli National Hemophilia Center, The Amalia Biron Thrombosis Research Institute and The Sackler Medical School, Tel Aviv University, Tel-Hashomer, Israel.
Objectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.
Methods: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82).
Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease.
Haemophilia
December 2024
Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy.
Article Synopsis
International Society on Thrombosis and Haemostasis Clinical Practice Guideline for Treatment of Congenital Haemophilia-A Critical Appraisal.
Haemophilia
December 2024
Thrombosis and Hemostasis Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China.
Introduction: Evidence-based clinical practice guidelines drive optimal patient care and facilitate access to high-quality treatment. Creating guidelines for rare diseases such as haemophilia, where evidence does not often come from randomized controlled trials but from non-randomized and well-designed observational studies and real-world data, is challenging. The methodology used for assessing available evidence should consider this critical fact.
View Article and Find Full Text PDFAsciminib in Advanced-Line Treatment of Chronic Myeloid Leukemia.
Eur J Haematol
October 2024
School of Medicine, Faculty of Medicine and Health Sciences, Tel Aviv University, Tel Aviv, Israel.
Objectives: Asciminib, a novel allosteric BCR::ABL1 inhibitor, targets the ABL1 myristoyl pocket to potentially reduce toxicity and enhance efficacy. It is approved for Philadelphia chromosome-positive chronic-phase chronic myeloid leukemia (CML-CP) in patients with resistance or intolerance to two or more tyrosine kinase inhibitors (TKIs) or those with the T315I mutation.
Methods: This retrospective analysis evaluated patients with CML treated with asciminib under a managed-access program across eight Israeli centers from July 2019 to August 2022.
Exploring the wild almond, (Olivier), as a genetic source for almond breeding.
Tree Genet Genomes
September 2024
Fruit Tree Sciences, Volcani Center, Agricultural Research Organization, Newe Ya'ar Research Center, Ramat Yishay, Israel.
Article Synopsis
- The domestication of almonds has led to a significant loss of genetic diversity, narrowing the traits available for adaptation to changing environments.
- Modern breeding programs have further intensified this genetic bottleneck, primarily favoring a few successful almond cultivars used commercially worldwide.
- To combat these challenges, researchers created an interspecific F1 population between a cultivated almond and a local wild species, identifying and associating important traits to enhance almond genetic resources for future breeding efforts.
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