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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
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
Studying Cystic Fibrosis Treatments for People without Modulators
The purpose of this study is to collect health information and samples from people who have cystic fibrosis (CF). By doing this, researchers can learn more about CF and find new ways to treat it. We also want to help people with CF understand what it's like to be part of a research study. We are looking for volunteers who have CF and do not take a certain type of medicine called CFTR modulators.
Study Topics
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