Alnylam Pharmaceuticals

This study follows patients with a rare, inherited heart disease called ATTR amyloidosis with cardiomyopathy to see how safe and effective the drug vutrisiran is over a longer period. It includes about 700 people who either continued taking vutrisiran or switched to it from a sim…

This study is testing whether the drug patisiran can help people with ATTR amyloidosis, a condition where abnormal proteins build up and stiffen the heart muscle. It involves 360 participants who have heart failure symptoms from this disease. Researchers will measure if the drug …

This study is testing whether an investigational drug called vutrisiran can help people with a serious heart condition called ATTR amyloidosis with cardiomyopathy. The drug is given as an injection under the skin every three months. Researchers are comparing it to a placebo to se…

This study aims to better understand the natural course of Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder that causes kidney stones and kidney damage. Researchers will observe 207 patients already diagnosed with PH1 to see how the disease progresses over time in the …