Results 51 to 60 of about 1,680 (126)

Challenges and Opportunities in Post‐Marketing Reporting of Factor VIII Inhibitors With Efanesoctocog Alfa

open access: yes
Haemophilia, EarlyView.
Jennifer Dumont   +5 more
wiley   +1 more source

Large Language Models for Clinical Trial Protocol Assessments

open access: yesClinical Pharmacology &Therapeutics, Volume 119, Issue 2, Page 393-402, February 2026.
The purpose was to evaluate the utility of large language models (LLMs) for reviewing the statistical analysis plan (SAP) and pharmacokinetics–pharmacodynamics (PK–PD) components of clinical trial protocols. Clinical trial protocols and SAPs were obtained from clinicaltrials.gov for a testbed of 15 small‐molecule drugs, biologics, and global antibiotic
Euibeom Shin   +2 more
wiley   +1 more source

Understanding Treatment Care and Delays in Acquired Hemophilia A: A Population‐Based Study From Nova Scotia, Canada

open access: yeseJHaem, Volume 7, Issue 1, February 2026.
ABSTRACT Objectives Acquired Hemophilia A (AHA) is a rare, potentially life‐threatening bleeding disorder caused by autoantibodies against factor VIII (FVIII). We aimed to characterize patients with AHA in Nova Scotia, Canada, and assess diagnostic delays.
Rachelle Blackman   +4 more
wiley   +1 more source

Spotlight on emicizumab in the management of hemophilia A: patient selection and special considerations

open access: yesJournal of Blood Medicine, 2019
Koji Yada, Keiji NogamiDepartment of Pediatrics, Nara Medical University, Kashihara, Nara, JapanAbstract: Emicizumab is a factor (F)VIIIa-mimicking bispecific antibody recognizing FIXa and FX molecules.
Yada K, Nogami K
doaj  

Emicizumab use in major orthopedic surgery [PDF]

open access: yesBlood Advances, 2019
Key Points Major orthopedic surgery can be performed safely in hemophilia patients with inhibitors receiving emicizumab.
Craig D. Seaman, Margaret V. Ragni
openaire   +2 more sources

Interfacility Collaboration for Hemophilia Care in Japan: A Retrospective Database Study Using a Japanese Healthcare Claims Database

open access: yesHealth Science Reports, Volume 9, Issue 1, January 2026.
ABSTRACT Background and Aims In Japan, about 6000 patients with hemophilia A (PwHA) are separately cared for at ~1000 clinics or hospitals. Interfacility collaboration (IFC) between hemophilia treatment centers and “satellite” medical facilities located closer to patients is necessary to eliminate care disparities.
Ei Kinai   +2 more
wiley   +1 more source

Emicizumab utilization, safety, and outcomes in people with severe hemophilia and no inhibitors: 3-year follow-up. A report from the UK Haemophilia Centre Doctors’ Organisation

open access: yesResearch and Practice in Thrombosis and Haemostasis
Background: Emicizumab prophylaxis is restricted to severe hemophilia A in the UK. Treatment choice and safety remain a matter of debate. Objectives: This study was conducted to investigate factors influencing treatment choice, continued use, safety, and
Caroline Wall   +12 more
doaj   +1 more source

Rebalancing Hemostasis: Fitusiran as a First‐in‐Class RNAi Therapy in Hemophilia A and B

open access: yesHealth Science Reports, Volume 9, Issue 1, January 2026.
ABSTRACT Background and Aims Fitusiran is a first‐in‐class RNA interference (RNAi) therapy that lowers antithrombin (AT) to rebalance hemostasis in people with Hemophilia A or B, with or without inhibitors. Its recent FDA approval marks a major shift toward factor‐independent prophylaxis.
Raza Ur Rehman   +2 more
wiley   +1 more source

Implementing emicizumab in hemophilia inhibitor management: emicizumab should be prescribed after tolerance [PDF]

open access: yesBlood Advances, 2018
AbstractThis article has a companion Counterpoint by Le Quellec and Negrier.
openaire   +2 more sources

Balancing Promise and Peril: Hemophilia Gene Therapy Insights

open access: yesIUBMB Life, Volume 78, Issue 1, January 2026.
ABSTRACT Hemophilia is an inherited disorder characterized by impaired blood clotting caused by mutations in the genes responsible for producing coagulation factor (F) VIII (hemophilia A, HA) or FIX (hemophilia B, HB). Current treatment primarily relies on replacement therapy, involving frequent and costly infusions of FVIII or FIX concentrates.
Saicharan Akula   +4 more
wiley   +1 more source

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