Results from Expanded Access Programs: A Review of Academic Literature
ConclusionsBy summarizing characteristics of patients, diseases, and research methods described in all scientific literature published on expanded access, we provide a unique dataset for future research. We show that published scientific research on expanded access has surged over the past decades, partly due to COVID-19. However, international collaboration and equity in geographic access remain an issue of concern. Lastly, we stress the need for harmonization of research legislation and guidance on the value of expanded access data within real-world data frameworks  to improve equity in patient access and streamline fut...
Source: Drugs - May 18, 2023 Category: Drugs & Pharmacology Source Type: research

Baricitinib for the Treatment of Alopecia Areata
AbstractAlopecia areata (AA) is a relapsing, chronic, immune-mediated disease characterized by nonscarring, inflammatory hair loss that can affect any hair-bearing site. AA clinical presentation is heterogeneous. Its pathogenesis involves immune and genetic factors and several pro-inflammatory cytokines involved in AA pathogenesis, including interleukin-15 and interferon- γ, as well as Th2 cytokines, such as IL-4/IL-13, that signal through Janus kinase (JAK) pathway. AA treatment aims to stop its progression and reverse hair loss, and JAK inhibition has been shown to stop hair loss and reverse alopecia and has exhibited p...
Source: Drugs - May 17, 2023 Category: Drugs & Pharmacology Source Type: research

Trofinetide: First Approval
This article summarizes the milestones in the development of trofinetide leading to this first approval for Rett syndrome. (Source: Drugs)
Source: Drugs - May 16, 2023 Category: Drugs & Pharmacology Source Type: research