Ponta Delgada

The International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry is an ongoing global observational program tracking routine clinical outcomes in patients diagnosed with Gaucher disease. It includes patients regardless of their treatment status and aims to improve understanding of the variability, progression, and natural history of Gaucher disease. The Registry also seeks to support the medical community by developing monitoring recommendations, characterizing the patient population, and evaluating long-term treatment effectiveness of imiglucerase and eliglustat. The Registry involves no experimental treatments; patients receive clinical assessments and care as directed by their treating physicians. Additionally, there is a Gaucher Pregnancy Sub-registry that monitors pregnancy outcomes, complications, and infant growth up to 36 months postpartum for women with Gaucher disease. This Sub-registry collects medical and obstetric history and pregnancy data for participants who consent, without altering their standard care. Participants provide data through routine clinical visits, and researchers collect medical information to better understand patient outcomes and optimize care. The Registry tracks outcomes over long periods, including up to 42 years, to support ongoing care improvements. Women in the Pregnancy Sub-registry have additional data collected on pregnancy and infant growth, contributing to comprehensive monitoring of Gaucher disease impacts during and after pregnancy.

Researchers are evaluating how well seltorexant works and its safety as an added treatment to antidepressants in adults and elderly participants who have major depressive disorder with insomnia symptoms (MDDIS). The study focuses on people who have not responded adequately to current antidepressant therapy with selective serotonin reuptake inhibitors (SSRIs) or serotonin-norepinephrine reuptake inhibitors (SNRIs). This Phase 3 trial aims to assess the improvement of depressive symptoms and the maintenance effect of seltorexant compared to a placebo. Participants will receive either seltorexant or a matching placebo taken orally alongside their current antidepressant medication, which includes SSRIs or SNRIs. The study is divided into two parts: Part 1 evaluates changes in depression severity after 43 days, while Part 2 monitors the time to relapse for up to nearly three years in participants who achieved a stable response. Participants must continue their stable antidepressant dose during the study. During the study, participants will be assessed using the Montgomery-Asberg Depression Rating Scale to measure changes in depression symptoms and monitored for relapse over time. Safety and tolerability will also be evaluated throughout. The total participation includes an initial treatment phase and an extended maintenance phase, allowing researchers to understand both short-term and long-term effects of seltorexant as an adjunctive therapy.