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Studying the Medicine Migalastat for Children with Fabry Disease
Fabry disease is a rare genetic condition where the body cannot break down certain fats, causing them to build up and damage organs like the heart, kidneys, and nerves. The purpose of this study is to learn more about the medicine migalastat for this condition. Our researchers want to see if it is safe and helpful for children.
Study Topics
Genetic Disorders Rare Disease Children, Infants, Teen Health
Looking at Arm Size Differences in Kids with a Certain Type of Elbow Fracture
The purpose of this study is to gather information that might help us create a more accurate way to measure arm swelling when a child fractures their elbow. Using the same way to measure swelling might help doctors make decisions on treatment that can impact how kids heal from these types of injuries.
Study Topics
Helping Young Cancer Survivors Feel Better Using a Digital Tool
The purpose of this study is to learn more about a new digital tool to help adolescents and young adult cancer survivors manage their symptoms of depression. People in the study will use the tool, answer questions, and join a video call. The goal is to find out if the tool helps them manage their feelings better. All study visits and consultations will be done virtually.
Study Topics
Mental Health Cancer Cancer: Children, Teens Children, Infants, Teen Health
Studying Letermovir in Children and Teens After Kidney Transplant
The purpose of this study is to learn more about a medicine called letermovir among children and teens who have had a kidney transplant. Researchers want to see how the medicine works in the body and if it is safe. This will help doctors find the right dose for young patients.
Study Topics
Tracking the Long-Term Safety of the Medicine AGAMREE
Duchenne muscular dystrophy (DMD) is a disease that makes muscles get weaker over time. The purpose of this study is to track the long-term safety of a medicine called AGAMREE for this disease. People in the study will have yearly checkups and answer questions about their health. We are looking for volunteers who have DMD and are currently taking AGAMREE.
Study Topics
Genetic Disorders Men's Health Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
Using Mavacamten to Treat Adolescents with Cardiomyopathy
Obstructive Hypertrophic Cardiomyopathy (HCM) is a heart condition that makes it hard for the heart to pump blood and may make a person feel weak and tired. The purpose of this study is to see if the study drug, mavacamten, helps young people with obstructive HCM.
Study Topics
Studying the Medicine Apitegromab for Babies with Muscle Weakness
Spinal muscular atrophy is a disease that makes muscles weak and hard to move. The purpose of this study is to test a new medicine called apitegromab. We want to see if it is safe and helpful for babies under 2 years old who still have trouble moving even though they already got another treatment called survival motor neuron therapy.
Study Topics
Genetic Disorders Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
Learning More About Chronic Kidney Disease
The purpose of this study is to find out what helps kidneys stay healthy and what makes them stop working well. Our researchers will look at how kidney problems can affect the brain, behavior, heart, growth, and overall health. We hope to learn more about how to make medical care better for kids, teenagers and young adults.
Study Topics
Studying New Medicines for Inflammatory Bowel Disease
Inflammatory Bowel Disease is a condition that causes swelling and pain in the digestive tract, making it hard for your body to digest food normally. The purpose of this study is to compare new medicines for this condition. Our researchers want to see if the medicines are safe and helpful for people who did not get better with other treatments.
Study Topics
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
Testing Seizure Medicine for Kids
The purpose of this study is to test a liquid medicine called zonisamide for kids with seizures. We want to see how the medicine moves in the body and if it is safe. The study is for kids aged 1 month to 17 years who have partial-onset seizures.
Study Topics
Using CTX001 to Treat Severe Sickle Cell Disease in Children
The purpose of this study is to learn more about using the study drug, CTX001, to treat sickle cell disease in children. We want to see how effective it is and if there are any side effects.
Study Topics
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
Comparing Treatments in Children Newly Diagnosed with Crohn's Disease
The purpose of this study is to learn more about why some children with Crohn's disease heal their bowel inflammation after one type of treatment and why others do not. Volunteers must have suspected Crohn's disease.
Study Topics
Collecting Data from Hemophilia Patients
The purpose of this study is to collect data on a blood disorder called hemophilia. The data will be used to improve the care of patients.
Study Topics
Heart Health Cancer Cancer: Children, Teens Children, Infants, Teen Health
Study to See How Well the Study Drug EPX-100 Works for Treating Dravet Syndrome
The purpose of this study is to see how well the study drug, EPX-100, works for treating Dravet syndrome. We also want to learn more about possible side effects.
Study Topics
Learning More About Treating Childhood Absence Epilepsy and Juvenile Absence Epilepsy with Brivaracetam
The purpose of this study is to see how well the study drug, brivaracetam, treats childhood absence epilepsy or juvenile absence epilepsy. We also want to learn more about any possible side effects it may cause.
Study Topics
Using Technology to Improve Care for Teens with Inflammatory Bowel Disease
Inflammatory bowel disease (IBD) affects the digestive system. Types of IBD include Ulcerative Colitis and Crohn's disease. The purpose of this study is to find out if text messaging can be used to help care for people with IBD. Volunteers must have a smartphone with text messaging.
Study Topics
High Blood Pressure in Children and Teenagers
The purpose of this study is to understand what causes high blood pressure in young people. We also want to learn more about how high blood pressure can cause damage to the heart and kidneys.
Study Topics
Healthy Volunteers Heart Health Children, Infants, Teen Health
Comparing Ways to Improve Blood Flow in Newborns' Lungs
The purpose of this study is to looks at two different methods to help increase blood flow in the lungs of newborn babies. One method uses a shunt, which is a small tube, and the other uses a stent, which is a tiny mesh-like device. We want to learn if one of the two options is better than the other.
Study Topics