Shire

This study tests a drug called SHP611, given as a spinal injection, in 36 children with late infantile metachromatic leukodystrophy (MLD), a rare and severe brain disease. The main goal is to see if the treatment helps children keep their ability to walk and move for as long as p…

This study followed 24 people with metachromatic leukodystrophy (MLD), a rare and serious brain disease, who were already receiving an experimental drug called HGT-1110. The main goal was to check for side effects and immune reactions over a long period. Researchers wanted to see…

This study followed 21 boys with Hunter syndrome who started taking Elaprase before age 6. Researchers tracked their height and weight for at least 5 years to see if the drug helps them grow more like other children. The study also monitored safety and compared growth data to unt…

This study followed 140 people with hereditary angioedema (HAE) who were already taking lanadelumab. Researchers compared the number of swelling attacks during treatment to the number before starting the drug. Participants reported their symptoms via a smartphone app and during c…

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

This study checked how well patients and doctors understand the risks and safe use of NATPARA, a drug for hypoparathyroidism. About 200 people took a survey online, by phone, or on paper. No medicine was given—the goal was just to measure knowledge and attitudes.

This early-stage study tested a single dose of the drug maribavir in 24 healthy adults—12 of Japanese descent and 12 of non-Hispanic Caucasian descent. The goal was to see if the drug behaves differently in these groups and to check safety. Participants took different doses to un…