Dundonald

Researchers are studying participants with Congenital Myasthenic Syndromes (CMS) caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. The goal is to understand symptoms, quality of life, and disease activity in these patients over time. This natural history study gathers important information about how CMS affects participants and tracks changes in their health. Participants will attend up to four study visits during which clinical assessments are performed. These assessments focus on evaluating symptoms and overall quality of life related to CMS. No specific treatments or interventions are administered, as the study is observational and aims to collect detailed data about the condition's progression. Throughout the study, researchers collect data retrospectively and prospectively over a period of up to 12 months. This includes information on diagnosis, healthcare use, medications, and changes in health status related to CMS. The study carefully monitors participants to better characterize the natural course of the disease and its impact on daily living.

Researchers are evaluating the effectiveness and safety of the investigational drug omecamtiv mecarbil in patients with symptomatic heart failure and severely reduced ejection fraction. This study aims to see if the drug can lower the risk of serious heart-related events, such as cardiovascular death, heart failure events, need for heart device implantation, heart transplantation, or stroke. The study is a Phase 3, multi-center, double-blind, randomized, placebo-controlled trial involving adults aged 18 to 85 years. Participants will be randomly assigned to receive either omecamtiv mecarbil or a placebo, both given as oral tablets. The study is event-driven and will continue until at least 850 participants have experienced either a heart failure event or cardiovascular death. An interim analysis will be done after about 570 such events have occurred. The estimated participation period for each patient is up to 3 years. During the study, participants will be monitored for the time until the first occurrence of cardiovascular death or heart failure event. Researchers will collect various health data and monitor safety throughout the study. The study includes regular assessments to track heart function and adverse events, with follow-up until the study concludes when the required number of events is reached.