Results 61 to 70 of about 18,669 (233)

Safety of Biological Medicinal Products for Hereditary Coagulopathies: An Analysis of the Russian Pharmacovigilance Database

Безопасность и риск фармакотерапии
INTRODUCTION. With the expanding product range and the introduction of novel biological medicinal products for haemophilia A, haemophilia B, and von Willebrand disease in the Russian pharmaceutical market, post-marketing safety surveillance becomes ...
D. S. Fokina, O. V. Zhukova, A. L. Khokhlov   +2 more
doaj   +1 more source

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 more
wiley   +1 more source

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مجلة علوم ذي قار, 2019
This study aims to diagnosis some of the sickness cases by (Haemophilia) on some patients in three different healthy centers in al-Dewanya city . For such purpose , fifty cases of sickness by such disease are collected and laboratory test on them are ...
الاء محمد حسون, وجدان ثامر مهدي, وداد عبد جواد   +2 more
doaj   +4 more sources

The impact of severe haemophilia and the presence of target joints on health-related quality-of-life

Health and Quality of Life Outcomes, 2018
Background Joint damage remains a major complication associated with haemophilia and is widely accepted as one of the most debilitating symptoms for persons with severe haemophilia.
Jamie O’Hara, Shaun Walsh, Charlotte Camp, Giuseppe Mazza, Liz Carroll, Christina Hoxer, Lars Wilkinson   +6 more
doaj   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community

Haemophilia, 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, Sterre P. E. Willems, Saskia E. M. Schols, Rosanna Asselta, Gillian Lowe, Neil V. Morgan, Paula D. James   +6 more
wiley   +1 more source

Rare Bleeding Disorders and Bleeding Disorder of Unknown Cause: Current Understanding and Recent Developments

Haemophilia, EarlyView.
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini, Robert Klamroth, Bauke Haisma, Dino Mehic, Saskia E. M. Schols   +4 more
wiley   +1 more source

Patient Preferences for the Treatment of Haemophilia A and B in France

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia impacts health and quality of life. Advances in treatments such as factor replacement therapy (FRT), bispecific monoclonal antibody (BS‐mAb), gene therapy (GT), rebalancing treatment and high sustained factor necessitate understanding patient preferences to inform therapeutic strategies. Aim This study quantified trade‐
Yesim Dargaud, Yasmine Fahfouhi, Monia Ezzalfani, Astrid Foix Colonier, Alaeddine Sidhom, Aline Gauthier, Jérémie Rudant, Meriem Kessouri, Nicolas Giraud, Benoît Guillet, Anaïs Reynaud   +10 more
wiley   +1 more source

Real‐World Outcomes of Prophylaxis With rIX‐FP in Germany: A Prospective, Non‐Interventional Study in Haemophilia B

Haemophilia, EarlyView.
ABSTRACT Introduction Efficacy and safety of albutrepenonacog alfa (rIX‐FP) in people with haemophilia B (PwHB) has been previously demonstrated in clinical trials. However, real‐world data insights are required. Aim To describe real‐world effectiveness and tolerability of rIX‐FP in PwHB in Germany.
Johannes Oldenburg, Swee Wenning, Susanne Holzhauer, Wolfgang Miesbach, Robert Klamroth, Thomas Linhoff, Patrick Sommerer, Christian Pfrepper, Martin Olivieri, all study investigators   +9 more
wiley   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source