Results 111 to 120 of about 24,516 (282)

Add‐On Telitacicept Significantly Improves Outcome of Patients With Refractory Ocular Myasthenia Gravis a Real‐World Case Series

Brain and Behavior, Volume 16, Issue 1, January 2026.
6/7 patients achieved CMI by the third follow‐up, sustained to the fourth follow‐up, and 4/6 reached MSE by the fifth follow‐up. First real‐world evidence of adding telitacicept efficacy in refractory ocular MG symptoms Abstract Introduction Refractory ocular myasthenia gravis (MG) represents a significant therapeutic challenge, as conventional ...
Jing Lin, Yue Li, Mengcui Gui, Bitao Bu, Zhijun Li   +4 more
wiley   +1 more source

Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria.

PLoS ONE, 2020
BackgroundEculizumab has transformed management of paroxysmal nocturnal hemoglobinuria (PNH) since its approval. However, its biweekly dosing regimen remains a high treatment burden.
John Devin Peipert, Austin G Kulasekararaj, Anna Gaya, Saskia M C Langemeijer, Susan Yount, F Ataulfo Gonzalez-Fernandez, Emilio Ojeda Gutierrez, Christa Martens, Amy Sparling, Kimberly A Webster, David Cella, Ioannis Tomazos, Masayo Ogawa, Caroline I Piatek, Richard Wells, Flore Sicre de Fontbrune, Alexander Röth, Lindsay Mitchell, Anita Hill, Karen Kaiser   +19 more
doaj   +1 more source

Complement C3dg-mediated erythrophagocytosis: Implications for paroxysmal nocturnal hemoglobinuria [PDF]

, 2015
The clinical management of paroxysmal nocturnal hemoglobinuria (PNH), a rare but life-threatening hematologic disease, has fundamentally improved with the introduction of a therapeutic that prevents complement-mediated intravascular hemolysis. However, a
Koutsogiannaki, Sophia, Lambris, John D, Lin, Zhuoer, Ricci, Patrizia, Ricklin, Daniel, RISITANO, ANTONIO MARIA, Schmidt, Christoph Q.   +6 more
core   +1 more source

MUC4 mutations promote a thrombotic phenotype in patients with paroxysmal nocturnal haemoglobinuria by increasing the deposition of terminal complement

Clinical and Translational Medicine, Volume 16, Issue 1, January 2026.
1. PNH patients lack the complement regulatory proteins CD55 and CD59, triggering excessive activation of the terminal complement pathway and a prothrombotic state. 2. MUC4 mutation is an independent thrombotic risk factor in PNH. 3. Mutation/down‐regulation of MUC4 promotes the abnormal deposition of excessively activated terminal complement ...
Yingying Chen, Mengting Che, Chaomeng Wang, Qiaoyi Bronte Zhang, Weixin Chen, Hui Liu, Chunyan Liu, Guang Sheng Ling, Rong Fu   +8 more
wiley   +1 more source

Development and Pretesting of a Questionnaire to Assess Patient Experiences and Satisfaction with Medications (PESaM Questionnaire) [PDF]

, 2017
Background: The aim of this study was to develop, together with the Lung Foundation Netherlands and Dutch Kidney Patients Association, patients and clinicians, a measure to evaluate patient experiences with the orphan drugs pirfenidone (for idiopathic ...
Dirksen, C.D. (Carmen), Gelens, M., Kar, N.C. (Nicole) van de, Kimman, M.L. (Merel), Mostard, R. (Rémy), Rotteveel, A.H. (Adrienne H.), Storm, M. (Marjolein), Tak, N.C. (Nelleke C.), van Jaarsveld, X. (Xana), Wetzels, J.F.M. (Jack), Wijnsma, K.L. (Kioa L.), Wijsenbeek-Lourens, M.S. (Marlies)   +11 more
core   +1 more source

The Complement System in Post‐Transplant Kidney Injury

Organ Medicine, EarlyView.
Mengsi Hu
wiley   +1 more source

Delayed‐Onset Hemolysis in a Case of Hemolytic Uremic Syndrome: A Diagnostic Challenge

Case Reports in Hematology, Volume 2026, Issue 1, 2026.
Background Hemolytic uremic syndrome is a rare thrombotic microangiopathy characterized by hemolytic anemia, thrombocytopenia, and acute kidney injury. While commonly reported in children, adult‐onset presentations are less frequent and often atypical, leading to diagnostic delays.
Muhammad Younas, Noor Fatima, Zimal Naveed, Syeda Nafisa, Ana Antun   +4 more
wiley   +1 more source

Síndrome Hemolítico Urémico Atípico en Cuidados Intensivos: Caso Clínico de un Adulto [PDF]

, 2016
info:eu-repo/semantics ...
Agudo, I, Azevedo, L, Bento, L, Cavaco, R, Germano, N, Souto-Moura, T   +5 more
core   +1 more source

Eculizumab [PDF]

British Journal of Clinical Pharmacology, 2009
Eline A, Dubois, Adam F, Cohen
openaire   +2 more sources

Stratification of responders towards eculizumab using a structural epitope mapping strategy [PDF]

, 2016
The complement component 5 (C5)-binding antibody eculizumab is used to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS).
Berglund, Magnus M., Hu, Francis Jingxin, Nordling, Erik, Rockberg, Johan, Strömberg, Patrik, Uhlén, Mathias, Volk, Anna-Luisa   +6 more
core   +1 more source