Olivenza

Researchers are studying the long-term safety of TEGSEDI (inotersen) in adults with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN), a rare inherited disease where abnormal proteins build up in nerves and organs. This disease progresses over time and can be fatal. The study aims to observe patients in real-world settings to better understand how safe TEGSEDI is over a period of 10 years. The study is non-interventional and includes participants from Europe, the US, and Canada who receive care at specialized centers. The study does not require any mandatory visits, tests, or treatments beyond participants' usual clinical care. Data are collected when patients enroll and during their regular follow-up visits at their clinical sites. There are two groups: those who have recently taken TEGSEDI within 25 weeks before joining the study, and those who have not taken TEGSEDI recently but are eligible for it and may be receiving other treatments for hATTR-PN. Participants will be monitored over a long period to track safety and treatment outcomes. Data collection aligns with routine clinical visits without added procedures. The main outcome is to further understand the long-term safety of TEGSEDI under normal care conditions, with follow-up planned for up to 10 years. Patients provide informed consent before joining, and their health status is observed as part of their standard care.

Researchers are evaluating the safety and effectiveness of tezepelumab in children aged 5 to under 12 years who have severe uncontrolled asthma. These children must be on medium to high doses of inhaled corticosteroids along with at least one other asthma controller medication, with or without oral corticosteroids. This phase 3, multicenter, double-blind, placebo-controlled study aims to better understand how tezepelumab affects asthma control in this pediatric population. Participants will be randomly assigned in a 2:1 ratio to receive either subcutaneous injections of tezepelumab or a matching placebo for 52 weeks during the double-blind treatment period. Before this, there is a 4 to 6 week screening and run-in phase. After the treatment period, a 12-week follow-up phase occurs without treatment. Eligible participants can then join an optional open-label extension, receiving tezepelumab for an additional 104 weeks followed by another 12-week post-treatment follow-up. Throughout the study, participants will have regular assessments including lung function tests, asthma control questionnaires, and monitoring for asthma exacerbations. Researchers will measure the annualized rate of severe asthma flare-ups from the start of treatment to week 52. Safety and treatment adherence will also be closely monitored during all study phases, with total participation potentially extending over two years for those in the extension period.