Immedica Pharma Ab

This phase 3 trial is testing whether pegzilarginase, an enzyme replacement therapy, can lower plasma arginine levels and improve mobility in children and adults with arginase 1 deficiency, a rare genetic condition that causes high arginine levels and neurological problems. Parti…

This phase 1 trial is testing an investigational drug called AEB1102 in adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has returned or stopped responding to standard treatments. AEB1102 is a modified human enzyme that breaks down arginine, an amin…

This early-stage trial is testing an experimental drug called AEB1102 in people with advanced solid tumors that have not responded to standard treatments or for which no standard treatment exists. The main goal is to find the highest dose that is safe and to understand the drug's…

This study looked at the long-term safety of a medication called Chronocort in 76 people aged 16 and older with congenital adrenal hyperplasia (CAH), a condition where the body can't make enough cortisol. Researchers monitored for signs of over- or under-treatment, adrenal crises…

This study tested a weekly injection of pegzilarginase (Loargys) in 3 infants under 24 months old with arginase 1 deficiency, a rare genetic disorder that causes harmful buildup of arginine. The goal was to see if the drug is safe and can lower arginine levels in the blood. The t…