Alexion Pharmaceuticals, Inc.

This early study tested a drug called bertilimumab in 11 adults aged 60 and older with newly diagnosed bullous pemphigoid, a disease that causes painful blisters. The main goals were to check safety and see if the drug could reduce blistering. While results may guide future treat…

This study tested the drug eculizumab in 15 Chinese patients with refractory generalized myasthenia gravis (gMG), a condition causing severe muscle weakness that doesn't respond well to standard treatments. The goal was to see if eculizumab could improve daily activities and musc…

This study tested a new synthetic parathyroid hormone analog (AZP-3601) in healthy volunteers and patients with chronic hypoparathyroidism, a condition where the body doesn't produce enough parathyroid hormone. The main goal was to check safety and how the drug moves through the …

This study tested a drug called eculizumab in 57 people with severe Guillain-Barré syndrome (GBS), a rare condition where the immune system attacks nerves, causing muscle weakness and paralysis. Participants received either eculizumab or a placebo alongside standard treatment. Th…

This study followed 120 people with PNH, a rare blood disease, who switched from an older drug (eculizumab) to a newer one (ravulizumab). Researchers checked if ravulizumab kept their disease under control by measuring blood markers and symptoms. The goal was to see how well the …

This study tested a drug called ALXN2060 in 25 Japanese patients with a heart condition known as ATTR-CM, which causes heart failure. The goal was to see if the drug could improve how far patients could walk in 6 minutes and reduce heart-related hospital stays. Patients took the …

This study tested a drug called eculizumab in 25 children and adults in China with a rare blood and kidney disease called aHUS. The goal was to see if the drug could improve blood counts and kidney function. Participants received the drug and were monitored for safety and effecti…

This study tested a drug called ravulizumab in 41 children who developed a serious blood clotting condition (TMA) after a stem cell transplant. The goal was to see if the drug could help normalize blood counts and kidney function. The treatment lasted 26 weeks, and children were …

This study tested a drug called ravulizumab in 58 adults with neuromyelitis optica spectrum disorder (NMOSD), a rare condition where the immune system attacks the nerves, causing vision loss and paralysis. The goal was to see if the drug could prevent relapses (new attacks). Part…

This study tested a drug called ravulizumab in 148 people aged 12 and older who developed a serious blood clotting condition (thrombotic microangiopathy) after a stem cell transplant. The goal was to see if the drug could help patients live longer and avoid worsening of their con…

This study tested a drug called ravulizumab in 18 adults in China with a rare blood disease called PNH, which destroys red blood cells. The goal was to see if the drug could lower a key blood marker of cell damage and reduce the need for blood transfusions. Participants received …

This study collected data from over 6,000 people with PNH to monitor the safety of two drugs, Soliris and Ultomiris, and understand how the disease progresses. Participants included patients of any age who had a PNH diagnosis or a PNH clone. The goal was to track serious events l…

This study looked at how a high-fat, high-calorie meal changes the way the body absorbs acoramidis, a drug being developed for a heart condition. Eighteen healthy adults took the drug once with food and once without, and researchers measured drug levels in their blood. The result…