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Collecting Data on Patients with Cystic Fibrosis

The purpose of this study is to gather information on people with cystic fibrosis through a patient registry. The patient registry helps track important changes in people with cystic fibrosis. It also helps doctors understand the disease better, improve treatments, and help patients live longer.

Study Topics

Genetic Disorders Lung Health Children, Infants, Teen Health

Treating Hyperactivity in Children with Down Syndrome using Guanfacine

The purpose of this study is to learn more about how well the study drug, guanfacine, can treat hyperactivity in children with Down syndrome.

Study Topics

Genetic Disorders Children, Infants, Teen Health

Studying a Medicine for Babies with Alagille Syndrome

The purpose of this study is to learn more about a medicine called Odevixibat for babies with Alagille Syndrome. We want to see if this medicine can safely help reduce itching in babies with this health condition.

Study Topics

Genetic Disorders Rare Disease Children, Infants, Teen Health

Testing Genes for Cystic Fibrosis Diabetes

The purpose of this study is to understand why people with cystic fibrosis get diabetes. Both diseases are caused by changes in DNA and other molecules in the blood. These changes can cause thick mucus in the pancreas, which damages cells that make insulin, leading to diabetes.

Study Topics

Genetic Disorders Lung Health Children, Infants, Teen Health Diabetes