The purpose of this study is to see how teclistamab by itself compares to study treatment combinations that are commonly used to treat multiple myeloma. These study treatment combinations are either pomalidomide, bortezomib and dexamethasone (called PVd) or carfilzomib and dexamethasone (called Kd). The patients will be randomly assigned to one of the study…
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…
We are doing this study to learn if the MMprofiler test can help your doctor in making decisions about your treatment. The MMprofiler genomic test will look at a large number of normal genes. The test will predict risk of your multiple myeloma (MM) by assessing whether these genes are turned on or off in your cancer cells. The purpose of this research is to…
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
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…