Shire

This study followed 21 children with Hunter Syndrome who started treatment with Elaprase before age 6. Researchers measured changes in height and weight over time to see how the drug affected growth. The study also monitored safety and compared results to untreated patients from …

This study checked the long-term safety of a drug called HGT-1110 in 24 people with metachromatic leukodystrophy (MLD), a rare nerve disease. All participants had already received the drug in an earlier study. Researchers monitored side effects and immune responses over many year…

This study followed 140 people with hereditary angioedema (HAE) who were already taking lanadelumab to see how well it works in real life. Participants reported their swelling attacks using a smartphone app and during routine doctor visits for up to three years. The main goal was…

This study tested a long-acting non-stimulant medication called guanfacine (TAK-503) in 396 children and teenagers aged 6 to 17 with ADHD whose previous stimulant treatments did not work well. The study compared guanfacine to a placebo (inactive pill) and another ADHD drug (atomo…

This study looked at how the drug maribavir is absorbed and processed in healthy adults of Japanese descent compared to non-Hispanic Caucasian adults. Twenty-four volunteers received a single oral dose of maribavir, and researchers measured drug levels in the blood to check for d…

This study surveyed 200 patients and doctors to see how well they understand the risks and safe use of NATPARA, a drug for hypoparathyroidism. Participants answered questions online, by phone, or on paper. No study medicine was given. The goal was to measure knowledge, not to tre…