Creation of a pediatric robotic spine surgery registry will allow for data collection and analysis on the coupled use of robotics and navigation, as well as patient-specific rods in pediatric spine
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This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.
This
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The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein
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In this research study the investigators want to learn more about how well the investigators can visualize scar tissue in the heart by MRI. In patients with congenital heart disease who need a
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COMPASS is a prospective multicenter randomized interventional trial. Participants with ductal-dependent pulmonary blood flow will be randomized to receive either a systemic-to-pulmonary artery shunt
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This is a multi-center, non-interventional registry to create and maintain a database of participants to serve as a recruitment source for current and future DAIT NIAID-sponsored Childhood Asthma in
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The investigators have designed a 2-center, pilot feasibility, randomized controlled trial (PROXIMUS) to determine the feasibility and safety of a larger multi center, randomized open-label trial
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The main purpose of this study is to investigate efficacy, pharmacokinetics and safety of the drug in pediatric participants with moderately to severely active ulcerative colitis (UC).
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
All
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The goal of this clinical trial is to test the effects of a ketogenic diet on the progression and control of type 1 diabetes in children with newly diagnosed diabetes. The main questions to answer are
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This is a phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NST-6179 in subjects with
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Overall, this observational cohort study aims too:
1. Implement rapid trio WGS for all children presenting to our health systems with epilepsy onset under 12 months of age.
2. Utilize electronic
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Calcium Release Deficiency Syndrome (CRDS) is a novel inherited arrhythmia syndrome secondary to RyR2 loss-of-function that confers a risk of sudden cardiac death. Diagnosis of CRDS presently requires
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The goal of this study is to test a web-based psychoeducational resource for adolescents with central disorders of hypersomnolence and their families. The investigators hope to assess the website's
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The goal of this research study is to learn whether investigators can successfully give the PediRISE program to families-in other words, whether most families are interested in participating in a
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The goal of this observational prospective study is to determine the health impact of parenthood on United States (US) people with CF in the era of CF transmembrane regulator protein (CFTR) modulators
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The aim of this study is to compare the impact of common (standard of care) language vs positive language used by clinicians during inhalational induction of anesthesia on anxiety and negative
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This is a prospective, randomized, controlled, non-inferiority study of patients undergoing tonsil surgeries at Boston Children's Hospital Waltham. The overall aim is to evaluate the efficacy of an
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The VIBRANT study aims to validate biosensors to objectively and directly measure symptoms in Rett syndrome and create confidence in the use of these devices in clinical trials. VIBRANT will use
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