Results 31 to 40 of about 2,525 (118)

A phase III, randomised, double‐blind, multi‐national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria

eJHaem, 2023
Treatment of paroxysmal nocturnal haemoglobinuria (PNH) includes the monoclonal antibody eculizumab. This randomised, double‐blind, multi‐national cross‐over Phase III study in PNH patients aimed to demonstrate the equivalence of the proposed eculizumab ...
Jun Ho Jang, Roberta Demichelis Gomez, Horia Bumbea, Larysa Nogaieva, Lily Lee Lee Wong, Soo Min Lim, Younsoo Kim, Jihye Park   +7 more
doaj   +1 more source

Thromboembolic events and haematological diseases: a case of stroke as clinical onset of a paroxysmal nocturnal haemoglobinuria

Thrombosis Journal, 2004
Some haematological diseases are associated to an increased risk of thromboembolic events. We report a case of paroxysmal nocturnal haemoglobinuria (PNH) in which a cerebrovascular event represented the first clinical manifestation of disease.
Viggiano Vito, Castaldo Giampiero, Bonamassa Barbara, Di Micco Pierpaolo, Izzo Tiziana, Granata Gianluca, Picillo Ugo, Castaldo Giuseppe, Niglio Alferio   +8 more
doaj   +1 more source

DELAYED ERYTHROID AND PLATELET RESPONSE TO ECULIZUMAB IN PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA – A CASE REPORT AND LITERATURE REVIEW [PDF]

European Medical Journal Hematology, 2013
Paroxysmal nocturnal haemoglobinuria (PNH) is an acquired clonal disorder of haemopoiesis characterised by haemolytic anaemia, thrombophilia and variable cytopaenias.
Andres L. Brodsky, Laura B. Colin
doaj  

The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors: clinical outcomes and medical encounters from the patient perspective

Hematology, 2022
Objectives: To assess the clinical and healthcare resource burden among C5 inhibitor (C5i)-treated patients with paroxysmal nocturnal haemoglobinuria (PNH), using patient-reported data.Methods: This web-based, cross-sectional survey (01FEB2021–31MAR2021)
Flore Sicre de Fontbrune, Pascale Burmester, Maria Piggin, Joana E. Matos, Halley Costantino, Koo Wilson, Zalmai Hakimi, Jameel Nazir, Renaud Desgraz, Jesse Fishman, Emmelie Persson, Jens Panse   +11 more
doaj   +1 more source

Paroxysmal Nocturnal Hemoglobinuria (PNH): A Comprehensive Review

Haematology Journal of Bangladesh
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired clonal hemopoietic stem cell disorder characterized by complement mediated chronic intravascular haemolysis, bone marrow failure and life-threatening thrombosis.
Muhammad Kamruzzaman
doaj   +1 more source

A Case Report: Autoimmune Haemolytic Anaemia & Paroxysmal Nocturnal Haemoglobinuria Association

Haematology Journal of Bangladesh
Autoimmune haemolytic anaemia (AIHA) and paroxysmal nocturnal haemoglobinuria (PNH) are two distinct causes of haemolytic anaemia. They have different mechanisms that underpin their pathogenesis and, therefore, require different treatment strategies ...
Fatima- Tuz- Zohra, Abu Hasan Rubel, Mohd. Tariqul Islam, Md. Sirazul Islam   +3 more
doaj   +1 more source

Beyond Nature's Blueprint: Macrocyclic Peptides Containing Unnatural Amino Acids in Therapeutic Development

Angewandte Chemie International Edition, EarlyView.
Strategic incorporation of unnatural amino acids transforms macrocyclic peptides into drug‐like molecules capable of engaging challenging targets. These building blocks enhance stability, permeability, and bioavailability, accelerating the development of next‐generation peptide therapeutics.
Krishna K. Sharma, Komal Sharma, Anku Sharma, Kamya Rao, Rahul Jain, Brett VanVeller   +5 more
wiley   +1 more source

Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

Frontiers in Pharmacology, 2022
Rare diseases (RD) pose serious challenges in terms of both diagnosis and treatment. Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the previous neglect of RD, by providing incentives for development of “orphan drugs” (OD) for ...
Lucio Luzzatto, Lucio Luzzatto, Julie Makani   +2 more
doaj   +1 more source

Exploring individualized crovalimab dosing in PNH through in silico modelling: Potential for improved convenience and cost efficiency

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired haematopoietic stem cell disorder. Crovalimab, a complement C5‐inhibitor, is approved for PNH and can be self‐administered subcutaneously every 4 weeks, offering a more convenient route than intravenous C5‐inhibitors.
Mendy ter Avest, Simon E. Koele, Nicole C. A. J. van de Kar, Saskia M. C. Langemeijer, Rob ter Heine   +4 more
wiley   +1 more source

Venous Thromboembolism in Hematologic Malignancies: Incidence, Risk Factors, and the Role of Direct Oral Anticoagulants

European Journal of Haematology, EarlyView.
ABSTRACT Thrombotic events, particularly venous thromboembolism (VTE), are a significant source of morbidity and mortality among patients with hematologic malignancies. These patients face unique challenges due to treatment‐related complications such as thrombocytopenia, coagulopathy, and heightened bleeding risk.
Mario Biglietto, Silvia Sorella, Erminia Baldacci, Massimo Breccia, Sabina Chiaretti, Marcello Capriata, Livia Donzelli, Gianna Maria D'Elia, Salvatore Giacomo Morano, Ilaria Del Giudice, Maurizio Martelli, Cristina Santoro, Antonio Chistolini   +12 more
wiley   +1 more source