This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that ...
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
This is a single center, prospective, observational study to demonstrate the clinical validity of the Intrinsic LifeSciences (ILS) Intrinsic Hepcidin IDx™ Test in the diagnosis and management of iron ...
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood ...
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 ...
131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disease. In this pediatric phase 1 trial, 131I-MIBG will ...
Study Objectives The objectives for this randomized trial are: 1. To determine the efficacy of daily low-dose atropine (0.01%) for slowing myopia progression over a two-year treatment period in ...
This study is enrolling participants by invitation only. This is an open-label, safety extension study for subjects who participated in the ARC007 study.
Bridge-Enhanced ACL Restoration (BEAR) is a new procedure being developed to treat patients with ACL injuries. In the BEAR procedure, an implant is placed between the torn ends of the ACL and the ...
This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and currently receiving ...