Allergists and clinical immunologists around the world are increasingly faced with the task of
addressing drug allergy and hypersensitivity due to the increase in drug reactions …

We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

Inherited bleeding, thrombotic, and platelet disorders (BPDs) are diseases that affect∼ 300
individuals per million births. With the exception of hemophilia and von Willebrand disease …

Background Severe haemophilia is associated with major psychological and economic
burden for patients, caregivers, and the wider health care system. This burden has been …

Hemophilia A is an X-linked hereditary bleeding disorder due to the deficiency of
coagulation factor VIII (FVIII). According to the degree of FVIII deficiency, mild, moderate or …

Introduction Hemophilia comprises a group of X-linked hemorrhagic disorders that result
from a deficiency of coagulation factors. The disorder affects mainly males and leads to …

Against a background of a rapidly evolving treatment landscape, a contemporary, evidence‐
based consolidated understanding of mortality in people with congenital hemophilia A …

Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency
caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as …

We investigated the efficacy of liver-directed gene therapy using lentiviral vectors in a large
animal model of hemophilia B and evaluated the risk of insertional mutagenesis in tumor …

Hemophilia is a hematological disorder characterized by a partial or complete deficiency of
clotting factor VIII or IX. Its bleeding complications primarily affect the musculoskeletal …