We are looking at how infants and young children breathe in different positions. This will help us understand how children with different types of lung hypoplasia breathe and how the lung changes and grows. To do this, we will measure regional lung ventilation by electrical impedance tomography (EIT) in three positions: lying on the back, right- and left-side in children with pulmonary hypoplasia and in healthy children without lung disease. EIT has been well established as a safe, non-invasive, radiation free method of assessing regional lung ventilation. There is no compensation available for this study.

CHOP is conducting a research study that will help make it possible to do future studies in children and family members both with and without problems with their brain and nervous system. This study involving collecting blood and other types of samples from participants along with medical information. These samples and the information will be stored in a "repository" for an indefinite amount of time. We'll use these tools in the future to create and develop new research studies that may help us find answers to some of these difficult illnesses.

The purpose of this research study is to help us understand how well tofacitinib works as a treatment for ulcerative colitis or indeterminate colitis in pediatric patients. We are looking to enroll, males or females ages 6 to 21 years old with a diagnosis of ulcerative colitis or indeterminate colitis who have started tofacitinib therapy prescribed by their primary gastroenterologist within the past 2 weeks or will be starting tofafacitinib within 1 month.

This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver. In the liver is where factor VIII protein is made. The study product, SPK-8011, is available only to participants in this research study. This product has not been approved yet for use in the general public. This will be the first hemophilia A gene transfer research study done in the United States. To learn more about the benefits and risks related to this study, please contact Dr. Lindsey George at (267) 425-0132. In order to participate in this study, you must be male, 18 years or older, have hemophilia A with FVIII activity levels less or equal to 2%, a history of bleeding that requires prophylaxis or on demand therapy, and no history of inhibitor to factor VIII. This study requires a number of visits over the course of 12 months. All costs related to participation in the study will either be covered or reimbursed by the Sponsor of the study, Spark Therapeutics, and participants will be compensated for their time.

The purpose of this study is to see if a new investigational drug (Upadacitinib) is safe and well tolerated and also to determine the appropriate dose for subjects with polyarticular course juvenile arthritis (pcJIA). The study is a two part study; in Part 1 subjects will be categorized based on age group, and the dose administered for seven consecutive days will be based on four different body weight categories. Subjects who complete Part 1 and are benefiting from study drug with no ongoing adverse events of special interest or serious adverse events, based on investigator's clinical judgment and with subject/family's agreement, will have the option to enroll in Part 2 to receive open-label upadacitinib. Participants will remain in part 2 for up to 156 weeks. Researchers will also be testing the pharmacokinetics (PK) of the study drug, which looks at how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body. Subjects will be asked to come into the Rheumatology clinic for blood and urine testing, a joint exam, and administration of study drug.

Patients with who have had Fontan surgery are at risk for the development of liver fibrosis and cirrhosis. New MRI based imaging techniques allow for non-invasive assessment of liver stiffness and fibrosis. These methods have been used in other diseases, but have not been studied in those who have undergone the Fontan operation. In this study we will test the feasibility of MRI based methods for the assessment of liver disease, and compare the results of these studies to those obtained from clinical liver biopsy.

This is a randomized controlled trial that tests whether a program designed to maintain a high fluid intake decreases kidney stone recurrence. Patients 12 years of age or older who have had a kidney stone in the last 3 years may be eligible.

To identify the underlying causes of bone marrow failure diseases for the purpose of developing and modifying treatment and diagnosis procedures.

This study is a trial comparing the effects of erenumab to placebo in children ages 6-17 years old with migraine 15 days per month or more. Eligible participants will be given either erenumab or placebo, assigned randomly (like the flip of a coin), followed by an open-label phase in which all participants receive erenumab. Erenumab has been FDA-approved for use in adults to decrease the frequency of migraine. Patients may be eligible to participate if they have had migraine for at least one year, and agree to come to CHOP's main hospital for several study visits. Patients whose headache started suddenly (New Daily Persistent Headache), patients whose headache started after a concussion (Post-Traumatic Headache), and patients with severe depression are not eligible for this study.

The purpose of this research study is to collect information over time in juvenile SpA and back pain patients. The information from this study will be used to better understand these conditions in children as well as improve treatment guidelines. The procedures include questionnaires, medical record review, and optional biospecimen contributions (blood).