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A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants with Hypophosphatasia (HPP)
This study collects observational Standard of Care (SOC) data in patients currently receiving asfotase alfa or about to start treatment with asfotase alfa as determined ...Phase N/A
Non-Interventional
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Environmental influences on Child Health Outcomes (ECHO) Cohort Data and Biospecimen Collection Protocol: The NYU Children's Health and Environment Study (NYU CHES) ECHO Cohort Study Site
ECHO is a nationwide research program whose mission is to enhance the health of children for generations to come.The goal is to learn how ...Phase N/A Non-Interventional
Accepts Healthy Volunteer
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The Prospective Natural History of Congenital Insensitivity to Pain with the Anhidrosis (CIPA)
The purpose of this study is to create a database of clinical information collected from you/your child’s medical chart on a yearly basis ...Phase N/A Non-Interventional
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Understanding Disease Burden in Biliary Atresia
It is our understanding that Biliary atresia (BA) can significantly affect a BA patient’s quality of life.? This study aims to assess the frequency ...Phase N/A Non-Interventional
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Collaborative Registry to Determine the Natural History of Barth Syndrome
The purpose of this research study is to collect data that we hope will help answer those questions and improve outcomes for children with Barth ...Phase N/A Non-Interventional
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SINGLE-CENTER OBSERVATIONAL STUDY OF SEIZURE TYPES IN RARE GENETIC EPILEPSIES
The purpose of this research study is to better describe specific movements that are seizures in rare genetic epilepsies (which may be different than other ...Phase N/A Non-Interventional
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FMT and Other Gut Microbial Therapies National Registry
A national data registry of patients receiving fecal microbiota transplantation (FMT) or other gut-related-microbiota products designed to prospectively assess short and long-term safety and effectiveness.Phase N/A Non-Interventional
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A single-center observational study of whole and regional brain volumes in patients with pediatric-onset demyelinating diseases
The purpose of this research study is to better understand how the brains of children and young adults with demyelinating diseases, such as multiple sclerosis ...Phase N/A Non-Interventional
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Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants with Alagille Syndrome (ALGS)
The purpose of this research study is to gather information on how well Bylvay (Odevixibat) works for treating Alagille syndrome using real-life data collected over ...Phase N/A Non-Interventional
