Alexion Pharmaceuticals, Inc.

This early-stage study tested a new hormone drug called AZP-3601 in healthy volunteers and people with chronic hypoparathyroidism, a condition where the body doesn't produce enough parathyroid hormone. Researchers wanted to understand the drug's safety, how it moves through the b…

This completed Phase 3 study tested whether the drug ravulizumab could effectively control paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disease, in Chinese adults who hadn't previously tried this type of medication. The trial involved 18 participants and me…

This study tested whether the drug eculizumab can control atypical hemolytic uremic syndrome (aHUS), a rare and serious blood disorder that can damage kidneys. The trial included 25 Chinese children and adults who had never received similar complement inhibitor treatments. Partic…

This study tested whether the drug eculizumab could help control severe myasthenia gravis in Chinese patients whose disease didn't respond to other treatments. Fifteen participants with significant muscle weakness received the drug for 26 weeks while researchers measured improvem…

This study tested a medication called ravulizumab in children who developed a dangerous blood clotting disorder (TMA) after a stem cell transplant. The goal was to see if adding this drug to standard care could help control the disorder by improving blood counts and kidney functi…

This Phase 3 clinical trial tested whether the drug ravulizumab could safely prevent relapses in adults with neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease that attacks the spinal cord and optic nerves. The study involved 58 participants and compared ou…

This study tested an experimental drug called ALXN2060 in Japanese patients with a serious heart condition caused by abnormal protein buildup. The main goals were to see if the drug could help patients walk farther and reduce their need for heart-related hospital visits. Twenty-f…

This small, early-stage study tested an experimental drug called bertilimumab in 11 older adults newly diagnosed with moderate to severe bullous pemphigoid, a painful autoimmune disease that causes large skin blisters. The main goal was to check the drug's safety and see if it co…

This study tested whether adding the drug eculizumab to standard treatment helps people with severe Guillain-Barré Syndrome (GBS) recover faster. GBS is a serious condition where the immune system attacks the nerves, causing muscle weakness and sometimes paralysis. The trial invo…

This study aimed to see if eating a high-fat meal changes how the body absorbs a drug called acoramidis. It involved 18 healthy adults who took the drug once with food and once on an empty stomach. Researchers measured drug levels in the blood to compare the two situations.

This registry study collected information from over 6,000 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The main goal was to monitor long-term safety of specific treatments and understand how the disease progresses over time. Researchers gathered…