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 ...
The objective of this study is to evaluate the safety and efficacy of sirolimus (0.2% and 0.4% formulations) and its vehicle when applied topically once daily for 12 weeks for the treatment of ...