Results 51 to 60 of about 1,680 (126)
Large Language Models for Clinical Trial Protocol Assessments
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
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
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]
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
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
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
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]
AbstractThis article has a companion Counterpoint by Le Quellec and Negrier.
openaire +2 more sources
Balancing Promise and Peril: Hemophilia Gene Therapy Insights
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

