This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
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 study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo
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The proposed study includes a newborn developmental intervention to improve neurodevelopmental (ND) and medical outcomes for infants with congenital heart disease (CHD) with improved parent well-being
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The purpose of this study is to learn more about changes in glucose levels in hospitalized infants with intestinal failure receiving parenteral nutrition or PN (nutrients delivered intravenously), as
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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 adult subjects
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The goal of this research study is to test the efficacy of a novel immunosuppressive agent, belumosudil, in allogeneic hematopoietic stem cell transplant (HSCT) recipients who have been newly
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The goal of this observational study is to identify candidate biomarkers in individuals with Rett Syndrome (RTT). The main questions it aims to answer are:
* Do these biomarkers change during
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Teleconsultation, or the use of video telecommunications technology to deliver expert recommendations for care remotely, has been used to improve the safety and quality of emergency care for children
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The goal of this clinical trial is to determine the impact of a parental support package for new trainee mothers which focuses on mentorship, lactation, and sleep, and access to perinatal care. The
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This is a trial in which 350 primary lung transplant recipients will be randomized (1:1) to receive either Tocilizumab (six doses over 20 weeks) plus standard triple maintenance immunosuppression or
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The goal of this study is to find the best way to help participants and families manage the stress of facing a serious illness and be better able to "bounce back" or be resilient after a difficult
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COMPRENDO (ChildhOod Malignancy Peer Research NavigatiOn) is a multi-site randomized clinical trial (RCT) that uses a Hybrid Type 1 design, to test the effects of a clinical intervention on patient
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This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF). Two hundred
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In this research study, the investigators want to learn more about the role of new innovative surgical devices, the Karl Storz Curved and Straight Fetoscopes for in-utero surgery. A fetoscope is like
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Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition. SDS-Like patients share clinical features with SDS
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This is a prospective multicenter biomarker study evaluating the prognostic impact of ctDNA detection at diagnosis in patients with Ewing sarcoma or osteosarcoma.
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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The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome.
The
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