Alexion Pharmaceuticals, Inc.

This study tested a new synthetic hormone called AZP-3601 in healthy volunteers and people with chronic hypoparathyroidism, a condition where the body doesn't produce enough parathyroid hormone. The goal was to check safety and how the drug works in the body. The trial involved 1…

This study tested a drug called ravulizumab in 58 adults with neuromyelitis optica spectrum disorder (NMOSD), a rare disease that causes inflammation in the nerves and spinal cord. The goal was to see if the drug could prevent or reduce relapses (sudden worsening of symptoms). Pa…

This study looked at 120 Italian patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched from the older drug eculizumab to the newer ravulizumab. Researchers tracked changes in a blood marker called LDH to see how well the new treatment controlled the disease in ever…

This study tested a drug called eculizumab in 15 Chinese adults with refractory generalized myasthenia gravis (gMG), a condition causing severe muscle weakness. The goal was to see if the drug could improve patients' ability to perform daily activities and reduce muscle weakness …

This study tested a drug called eculizumab in 25 children and adults in China with a rare kidney condition called atypical hemolytic uremic syndrome (aHUS). The goal was to see if the drug could improve blood counts and kidney function. Participants received the drug and were mon…

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 tested a drug called eculizumab in 57 people with severe Guillain-Barré syndrome (GBS), a rare nerve condition that can cause paralysis. Participants received either the drug or a placebo, along with standard treatment, to see if eculizumab helped them regain the abili…

This study tested a drug called ravulizumab in 41 children who developed a serious blood clotting problem (thrombotic microangiopathy) after a stem cell transplant. The treatment was given for 26 weeks, and researchers checked if it helped improve blood counts and kidney function…

This study tested a drug called bertilimumab in 11 adults aged 60 and older with newly diagnosed bullous pemphigoid, a condition that causes painful blisters. The goal was to see if the drug is safe and can reduce disease activity. Participants received the drug and were monitore…

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 improve survival and prevent the condition from getting wo…

This study tested a drug called ALXN2060 in 25 Japanese adults with a rare heart condition called ATTR-CM, which causes heart failure. The goal was to see if the drug helps patients walk farther, reduces hospital stays, and improves survival over 30 months. All participants recei…

This study looked at how a high-fat, high-calorie meal changes the way the drug acoramidis is processed in the body. 18 healthy adults took the drug once with food and once without, and researchers measured drug levels in their blood. The results will help decide if acoramidis sh…

This study collected health information from over 6,000 people with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The goal was to track the safety of two drugs, Soliris and Ultomiris, and understand how the disease progresses over time. Participants included b…