TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
The purpose of this research study is to determine if an investigational drug, VS-01, will help in the treatment of acute-on-chronic liver failure (ACLF) compared to supportive care (standard of therapy). ACLF is a potentially life-threatening condition characterized by a group of signs that occur together (syndrome) in patients with a late stage of…
Pain is a natural response to injury to let us know there may be damage to our bodies. Pain is the way our body tells us that we must take care of ourselves. Chronic pain is different and lasts for a long time. In some cases, chronic pain remains without any sign of body damage. Today, there is no way for a doctor to measure someone's chronic pain or…
The purpose of this study is to evaluate the efficacy and safety of ION-682884 given for 24 months in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) also known as Familial Amyloid Polyneuropathy (FAP), and determine if it can help people with mild or moderate polyneuropathy. To be in this study you must have FAP and be…
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…