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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
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.
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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.
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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
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
Combination Chemotherapy for Newly Diagnosed and Relapsed Wilms Tumor
The purpose of this study is to find out if we can improve treatment for those with Wilms tumors. We will be testing three different combinations of radiation and chemotherapy. The combination you receive will depend on your type of Wilms tumor.
Study Topics
Cancer Cancer: Kidney, Liver, Pancreas Children, Infants, Teen Health
Concussion Symptoms in Young People Ages 11-17
The purpose of this study is to learn more about concussion symptoms in young people ages 11 to 17. This may help us develop future treatments.
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A Study of Telehealth to Treat Children with Medical Complexity
The purpose of this study is to learn more about using telehealth visits for children with medical complexity. We want to find out if telehealth visits can improve the health of children with chronic health problems.
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Study of Selinexor for Treating High-Grade Glioma in Pediatric Patients
A glioma is a type of cancer that occurs in the brain or spine. Diffuse intrinsic pontine glioma is a type of high-grade glioma. The purpose of this study is to look at how well selinexor works when given to children and young adults with high-grade glioma or diffuse intrinsic pontine glioma. We also want to find the highest dose of selinexor that we can give safely along with radiation therapy.
Study Topics
Cancer Cancer: Brain, Nervous System, Spinal Cord Children, Infants, Teen Health
Studying an Exercise Program for Teens and Young Adults After Cancer Treatment
The purpose of this study is to find the best ways to help Hispanic or Latino/a teens and young adults who had cancer stay physically active at home. We will use a program that uses a Fitbit and social media to encourage exercise. The goal is to make sure the program fits the language and culture of the participants.
Study Topics
Cancer Cancer: Children, Teens Children, Infants, Teen Health
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.
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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.
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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
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.
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