Results 101 to 110 of about 92,747 (232)

Quizartinib in Combination With FLAG‐IDA for Relapsed or Refractory Acute Myeloid Leukemia (FLAG‐QUIDA): A PETHEMA Phase I‐II Trial

American Journal of Hematology, EarlyView.
ABSTRACT Quizartinib is a tyrosine kinase inhibitor with single agent activity in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) and has demonstrated efficacy in first‐line therapy when combined with intensive chemotherapy in both FLT3 ITD‐negative and positive AML.
Teresa Bernal, Javier Zambrano Márquez, Rebeca Rodríguez‐Veiga, Eduardo Rodríguez‐Arbolí, Juan Manuel Bergua‐Burgués, Susana Vives, Montserrat Arnán, Pilar Martínez Sánchez, Pilar Herrera‐Puente, Josefina Serrano‐López, Vicente Rubio, Inmaculada Marchante‐Cepillo, Marta Cervera‐Calvo, Andrés Novo García, Rosa Fernández Martín, Cristina Gil Cortés, Carlos Rodríguez‐Medina, Victor Noriega‐Concepción, Ana Alfonso‐Piérola, Carmen Loredo, María Antuña‐Casal, Blanca Boluda, Isabel Cano‐Ferri, Evelyn Acuña‐Cruz, Laura Torres‐Miñana, Joaquín Sánchez‐García, Cristina Bilbao, Maria Teresa Gómez‐Casares, David Martínez Cuadrón, Pau Montesinos   +29 more
wiley   +1 more source

Clinical Outcomes and Patient Experiences With Celiprolol Therapy in Vascular Ehlers–Danlos Syndrome: The First Non‐European Cohort

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Vascular Ehlers–Danlos syndrome (vEDS) is a hereditary connective tissue disorder caused by heterozygous pathogenic variants in COL3A1. European studies have shown that celiprolol may reduce the risk of life‐threatening vascular events, but outcomes in non‐European populations and the therapy's psychological impact remain unclear. We conducted
Megumi Furuhata‐Yoshimura, Tomomi Yamaguchi, Tomoki Kosho   +2 more
wiley   +1 more source

A randomized, placebo-controlled study of givosiran in patients with acute hepatic porphyrias (ENVISION): Final (36-month) analysis of the Taiwan Cohort

Journal of the Formosan Medical Association
Background/purpose: Acute hepatic porphyrias (AHP) are rare genetic disorders associated with acute neurovisceral attacks and chronic symptoms.
Ming-Jen Lee, Hung-Chou Kuo, Lin-Na Chou, Marianne T. Sweetser, Jiaan-Der Wang   +4 more
doaj   +1 more source

Histidine Supplementation Stabilizes Hearing and Vision and Improves Growth in HARS1‐Related Autosomal Recessive Disorder Associated With Usher‐Like Symptoms

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Autosomal recessive HARS1‐related disorder (originally described as Usher syndrome type 3B) caused by a homozygous Y454S variant in the histidyl‐tRNA synthetase gene (HARS1) is characterized by progressive sensorineural hearing and vision loss and respiratory deterioration with risk for sudden death following febrile illnesses.
Victoria Mok Siu, Rosan Kenana, Rana Chakrabarti, Sarah D. P. Wilhelm, Joseph Andrews, Susan J. Leat, Christina Parker, Michael Miller, Leslie A. Nangle, Wendy McCaul, Ashfia Chowdhury, Natalie Hutchings, Ryan A. Adams, Lauren Guy, Mandy Rhody, Verena Juncal, Marisa I. Mendes, Desiree E. C. Smith, Gajja S. Salomons, Angelica A. Moresco, Daphne L. McCulloch, D. Holmes Morton, Ilka U. Heinemann, C. Anthony Rupar   +23 more
wiley   +1 more source

Dosing Interval Extension of Dupilumab in CRSwNP: Five‐Year Real World Outcomes

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis with nasal polyps (CRSwNP) is a persistent, often Type 2‐mediated inflammatory disease that markedly impairs quality of life. While dupilumab provides rapid improvement, there is limited evidence on long‐term outcomes beyond 2 years, and the clinical impact of dosing‐interval extension remains unclear.
Nicholas J. Campion, Dioni‐Pinelopi Petsiou, Florian C. Fally, Karina Berbalk, Noah F. Melamed, Aldine Tu, Christina Morgenstern, Fana Alem Kidane, Mohammed Zghaebi, Linda Liu, Minghao Pan, Tina J. Bartosik, Victoria Stanek, Katarina Gangl, Julia Eckl‐Dorna, Sven Schneider   +15 more
wiley   +1 more source

Differences in Biologic Clinical Trials for Chronic Rhinosinusitis With Nasal Polyps—Are We Comparing Apples With Oranges?

International Forum of Allergy &Rhinology, EarlyView.
Key Points In recent years, several biologics targeting Type 2 inflammation have been developed for treating chronic rhinosinusitis with nasal polyps (CRSwNP). These have been studied in registrational randomized controlled trials (RCTs), which vary in their patient populations, trial design, endpoints, geography, timing, or data‐handling processes ...
Marjolein Cornet, Peter W. Hellings, Martin Desrosiers, Martin Wagenmann, Richard Follows, Laura Walrave, Luz Adriana Jimenez, Lee Tombs, Dawn Edwards, Peter Howarth, Joseph K. Han   +10 more
wiley   +1 more source

Inflammatory Signatures Beyond Th1/Th2 Endotypes Provide Insights Into Postoperative Recurrence in Chronic Rhinosinusitis

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) represents a spectrum of inflammatory endotypes, driven by distinct T helper (Th) immune responses. The high rate of recurrent disease despite appropriate medical and surgical interventions highlights the need for improved characterization beyond the traditional type 2 and non‐type 2 phenotypes to better
Liyona Kampel, Narin N. Carmel, Shaun Edalati, Hen Chaushu, Forsan Jahshan, Yaniv Hadi, Timur Pestrikov, Leonor Leider Trejo, Nidal Muhanna, Avraham Abergel   +9 more
wiley   +1 more source

Systemic Corticosteroids in the Management of Sinonasal Disease: An Evidence‐Based Expert Review

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) is a prevalent, heterogeneous inflammatory disease associated with significant morbidity. Systemic corticosteroids (SCS) are commonly prescribed for their anti‐inflammatory effects, but cumulative exposure carries risks, including metabolic, cardiovascular, and skeletal complications.
Andrew Thamboo, Deanna Gigliotti, Eugenio De Corso, Stella Lee, Benjamin S. Bleier, Christopher J. Chin, Marie‐Noelle Corriveau, Valerie Hox, Claire Hopkins, Peter H. Hwang, Arif Janjua, Ludger Klimek, Daniel Lee, John Lee, Amber Luong, Geoffrey Mortuaire, Joaquim Mullol, Zara M. Patel, John Scott, Doron Sommer, Leigh Sowerby, Marc Tewfik, Beatrice Voizard, Martin Wagenmannm, Sarah K. Wise, Jonathan Yip, Quirina Thompson, Martin Desrosiers, Yvonne Chan   +28 more
wiley   +1 more source

Chronic Rhinosinusitis and Nasal Polyps in Eosinophilic Granulomatosis With Polyangiitis: A Scoping Review

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) is one of the most common manifestations of eosinophilic granulomatosis with polyangiitis (EGPA), often preceding the diagnosis of systemic vasculitis by several years. The sinonasal presentation of EGPA typically resembles CRS with nasal polyps (CRSwNP), making it clinically indistinguishable from those
Alisha Sharma, Alyssa Y. Li, Teruko Kishibe, Dennis Wong, Kevin M. Sanders, Maggie Jiang, Heedo Lee, Christine Song, Kieran Mcintyre, Christian Pagnoux, Yvonne Chan, John M. Lee, Daniel J. Lee   +12 more
wiley   +1 more source

Biomimetic Nanoparticle Delivering Colchicine to Target Endothelial Inflammation for Atherosclerosis Treatment

Advanced NanoBiomed Research, EarlyView.
Platelet membrane‐coated layered double hydroxide nanoparticles were engineered to target inflamed endothelium at early‐stage atherosclerosis and enhance therapeutic efficacy. The biomimetic platform improves stability, immune escape, and inflamed vascular cell targeting, resulting in reduced oxidative stress and downregulated adhesion molecules.
Xiuwen Zhang, Lingqing Zong, Ashish Misra, Sanjay Patel, Zi Gu   +4 more
wiley   +1 more source