Shire

This study looked at the long-term safety of a drug called HGT-1110 for people with metachromatic leukodystrophy (MLD), a rare and serious brain disease. It included 24 people who had already been in an earlier study of the same drug. The main goal was to track side effects and s…

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 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 looked at whether a medication called guanfacine (TAK-503) can help children and teenagers aged 6 to 17 with ADHD who did not get better with stimulant drugs. The study had two parts: first, participants were randomly assigned to receive guanfacine, another ADHD drug (…

This study surveyed 200 patients and doctors to check their understanding of the risks and safe use of NATPARA, a drug for hypoparathyroidism. Participants answered true/false questions online, by phone, or on paper. No study medicine was given. The goal was to see how well key s…

This study looked at how the drug maribavir is absorbed and processed in healthy adults of Japanese descent compared to non-Hispanic Caucasian adults. It also tested three different doses in Japanese participants to see if the drug levels in the blood change proportionally with d…