Results 201 to 210 of about 162,133 (299)

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Anticoagulation with the mechanistically distinct FXI/FXIa antibodies amrecibart (REGN9933A2) and cenvacibart (REGN7508Cat).

Blood
Chalothorn D, Kithcart AP, Marin E, Somersan-Karakaya S, Lai K, Cauwberghs F, Ackroyd J, Mohammadi K, Shrestha A, Ehrlich G, Rafique A, Chatterjee I, Saotome K, Franklin MC, Murphy AJ, Olson W, Olenchock BA, Herman GA, Gutstein DE, Sirulnik A, Yancopoulos GD, Morton LG.   +21 more
europepmc   +1 more source

Thrombin reprograms endothelial extracellular vesicles. [PDF]

Blood Adv
Kulkarni P, McCrae KR.
europepmc   +1 more source

Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community

Haemophilia, EarlyView.
ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein, Sterre P. E. Willems, Saskia E. M. Schols, Rosanna Asselta, Gillian Lowe, Neil V. Morgan, Paula D. James   +6 more
wiley   +1 more source

Aptamer-based DNA nanoswitches for multiplexed protein detection.

Chem Commun (Camb)
Kovvali S, Beckles CA, Chandrasekaran AR, Halvorsen K.   +3 more
europepmc   +1 more source

Chemical Synthesis and Functional Screening of an 800 000-Member Library Using a Peptide-Pairing Strategy. [PDF]

Angew Chem Int Ed Engl
Zarda A, Deng X, Will E, Emel T, Romero M, Heinis C.   +5 more
europepmc   +1 more source

Rare Bleeding Disorders and Bleeding Disorder of Unknown Cause: Current Understanding and Recent Developments

Haemophilia, EarlyView.
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini, Robert Klamroth, Bauke Haisma, Dino Mehic, Saskia E. M. Schols   +4 more
wiley   +1 more source

Clinical Pharmacokinetics and Pharmacodynamics of Marstacimab, an Anti-tissue Factor Pathway Inhibitor Monoclonal Antibody, in Adolescent and Adult Participants with Hemophilia. [PDF]

Clin Pharmacokinet
Nayak S, Suzuki A, Ravva P, Raje S.
europepmc   +1 more source

Current State of Moderate Congenital Haemophilia A and the Need for Preventive Treatment

Haemophilia, EarlyView.
ABSTRACT Introduction The severity of haemophilia A is classified by the degree of factor VIII (FVIII) deficiency, rather than by clinical manifestations. However, FVIII activity alone does not necessarily accurately reflect clinical severity such as bleeding tendency, and patients with mild‐to‐moderate haemophilia A can experience significant disease ...
Masahiro Takeyama, Atsuki Yamashita, Tomoko Yamaguchi, Takahiro Ito, Yui Sugawara, Akiko Ioka, Tazuko Tokugawa   +6 more
wiley   +1 more source

Reversal of the Anticoagulant Effect of Milvexian by 4-Factor PCC and rFVIIa in Healthy Participants: A Two-Part, Randomized, Crossover Study. [PDF]

J Cardiovasc Pharmacol
Dishy V, Sha S, Koshkina A, Tesfaye F, Plotnikov AN, Makimura H, Chintala M.   +6 more
europepmc   +1 more source