Results 41 to 50 of about 2,667 (188)

Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria

Haematologica, 2020
Eculizumab is first-line treatment for paroxysmal nocturnal hemoglobinuria (PNH); however, approximately 11%-27% of patients may experience breakthrough hemolysis (BTH) on approved doses of eculizumab.
Robert A. Brodsky, Régis Peffault de Latour, Scott T. Rottinghaus, Alexander Röth, Antonio M. Risitano, Ilene C. Weitz, Peter Hillmen, Jaroslaw P. Maciejewski, Jeff Szer, Jong Wook Lee, Austin G. Kulasekararaj, Lori Volles, Andrew I. Damokosh, Stephan Ortiz, Lori Shafner, Peng Liu, Anita Hill, Hubert Schrezenmeier   +17 more
doaj   +1 more source

Complement inhibition in Myasthenia – from basics to RCT data [PDF]

, 2023
Myasthenia gravis (MG) is the prototypic autoimmune neurological disorder causing fatiguable muscle weakness either limited to the ocular muscles or becoming generalised involving the limb and bulbar muscles.
Jacob, Saiju
core   +2 more sources

Efficacy and safety of the long-acting C5 inhibitor ravulizumab in patients with atypical hemolytic uremic syndrome triggered by pregnancy: a subgroup analysis

BMC Nephrology, 2021
Background Atypical hemolytic uremic syndrome (aHUS) triggered by pregnancy is a rare disease caused by dysregulation of the alternative complement pathway that occurs in approximately 1 in 25,000 pregnancies.
Anja Gäckler, Ulf Schönermarck, Vladimir Dobronravov, Gaetano La Manna, Andrew Denker, Peng Liu, Maria Vinogradova, Sung-Soo Yoon, Manuel Praga   +8 more
doaj   +1 more source

Real-world evidence from the Global aHUS Registry confirms the safety and effectiveness of switching to ravulizumab from eculizumab in patients with aHUS: a plain language summary [PDF]

Registry; Atypical hemolytic uremic syndrome; RavulizumabRegistre; Síndrome hemolític urèmic atípic; RavulizumabRegistro; Síndrome hemolítico urémico atípico; RavulizumabWhat is aHUS?
Al-Dakkak, Imad, Ariceta Iraola, Gema, Greenbaum, Larry, Schaefer, Franz   +3 more
core   +1 more source

Inhibition of Classical and Alternative Complement Pathway by Ravulizumab and Eculizumab [PDF]

Objective: To explore the feasibility of classical (CH50) and alternative (AH50) complement pathway activity as potential biomarkers for treatment guidance and monitoring during therapy with ravulizumab in patients with generalized myasthenia gravis (gMG)
Doksani, Paolo, Dusemund, Carla, Gerischer, Lea, Herdick, Meret, Hoffmann, Sarah, Lehnerer, Sophie, Lünemann, Jan D., Meisel, Andreas, Mergenthaler, Philipp, Mönch, Maximilian, Schröder‐Braunstein, Jutta, Stascheit, Frauke, Stein, Maike, Suboh, Amani, Wabnitz, Guido   +14 more
core   +2 more sources

Immediate and sustained terminal complement inhibition with ravulizumab in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder

Frontiers in Neurology
ObjectiveTo assess the pharmacokinetics and pharmacodynamics of the long-acting terminal complement 5 (C5) inhibitor ravulizumab in adults with anti-aquaporin-4 antibody-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD) in the phase 3, open-
Stephan Ortiz, Sean J. Pittock, Achim Berthele, Michael Levy, Ichiro Nakashima, Celia Oreja-Guevara, Celia Oreja-Guevara, Kerstin Allen, Yasmin Mashhoon, Becky Parks, Ho Jin Kim   +10 more
doaj   +1 more source

Risk-Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis [PDF]

Introduction: This study used network meta-analysis (NMA) to inform and compare the number needed to treat (NNT), number needed to harm (NNH), and cost per improved outcome (CPIO) associated with more recently approved treatments for anti-acetylcholine ...
Brauer, Edward, Chen, Xin, Du, Mandy, Gelinas, Deborah, Habib, Ali A, Phillips, Glenn, Qi, Cynthia Z, Sacca, Francesco, Smith, A Gordon, Wolfe, Gil I, Yang, Hongbo   +10 more
core   +1 more source

Guideline for the management of myasthenic syndromes [PDF]

, 2023
Myasthenia gravis (MG), Lambert-Eaton myasthenic syndrome (LEMS), and congenital myasthenic syndromes (CMS) represent an etiologically heterogeneous group of (very) rare chronic diseases.
Abicht, Angela, Chan, Andrew, Della Marina, Adela, Hagenacker, Tim, Hekmat, Khosro, Hoffmann, Hans-Stefan, Hoffmann, Sarah, Jander, Sebastian, Keller, Christian, Marx, Alexander, Meisel, Andreas, Melms, Arthur, Melzer, Nico, Müller-Felber, Wolfgang, Pawlitzki, Marc, Rückert, Jens-Carsten, Schneider-Gold, Christiane, Schoser, Benedikt, Schreiner, Bettina, Schroeter, Michael, Schubert, Bettina, Sieb, Jörn-Peter, Wiendl, Heinz, Zimprich, Fritz   +23 more
core   +1 more source

Prediction of Myasthenia Gravis Worsening: A Machine Learning Algorithm Using Wearables and Patient‐Reported Measures

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Myasthenia gravis (MG) is a rare disorder characterized by fluctuating muscle weakness with potential life‐threatening crises. Timely interventions may be delayed by limited access to care and fragmented documentation. Our objective was to develop predictive algorithms for MG deterioration using multimodal telemedicine data ...
Maike Stein, Haoqi Sun, Sophie Lehnerer, Lea Gerischer, Maximilian Mönch, Christian Meisel, Andreas Meisel, Pushpa Narayanaswami   +7 more
wiley   +1 more source

No evidence for hypogammaglobulinemia in patients with paroxysmal nocturnal hemoglobinuria (PNH) chronically treated with ravulizumab.

PLoS ONE, 2020
IntroductionRavulizumab (ALXN1210) is a long-lasting recycling IgG monoclonal antibody with an increased affinity for the neonatal Fc receptor (FcRn). The FcRn is essential for regulating IgG homeostasis. Saturation of the FcRn pathway is seen under high
Ferras Alashkar, Scott Rottinghaus, Colin Vance, Dörte Herich-Terhürne, Ulrich Dührsen, Roland Assert, Alexander Röth   +6 more
doaj   +1 more source