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View article: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders Open
Centralization and expansion of expertise and resources, flexible innovative research and regulatory approaches, and inclusion of all people with ultra-rare inherited BDs and their health care professionals will be essential to capitalize …
View article: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders Open
Ultra-rare inherited bleeding disorders (BDs) present important challenges for generating a strong evidence foundation for optimal diagnosis and management. Without disorder-appropriate treatment, affected individuals potentially face life…
View article: Pharmacodynamic Activity of the Novel Neurokinin-3 Receptor Antagonist SJX-653 in Healthy Men
Pharmacodynamic Activity of the Novel Neurokinin-3 Receptor Antagonist SJX-653 in Healthy Men Open
Context SJX-653 is a novel neurokinin 3 receptor (NK3R) antagonist. The NK3 pathway is a central regulator of gonadotropin releasing hormone (GnRH) secretion and has also been implicated in the generation of hot flashes. Therefore, decreas…
View article: Late breaking Abstracts
Late breaking Abstracts Open
Background: Damoctocog alfa pegol (BAY 94-9027, Jivi ® ) and rurioctocog alfa pegol (BAX 855, Adynovi ® ) are PEGylated, extended half-life, recombinant factor VIII (FVIII) products, indicated for use in patients ≥ 12 years of age with hae…
View article: SAT-LB073 SJX-653, a Novel Selective NK3 Antagonist, Demonstrates Activity on Pharmacodynamic Markers of NK3 Target Engagement
SAT-LB073 SJX-653, a Novel Selective NK3 Antagonist, Demonstrates Activity on Pharmacodynamic Markers of NK3 Target Engagement Open
Background: NK3 antagonism is a clinically validated mechanism of action for treating menopausal vasomotor symptoms (“hot flashes”). In men, reductions of LH and T are sensitive pharmacodynamic markers of central NK3 antagonism, due to the…