This is a registry study to evaluate the long-term safety and effectiveness of adalimumab in pediatric patients with moderately to severely active CD who are treated as recommended in the local ...
The primary objective of this study is to evaluate if Broncho-Vaxom® given to high risk infants for 10 days, monthly, for two consecutive years can increase time to occurrence of the first episode of ...
The purpose of this study is to determine the safety and maximum effective dose (MED) of Interleukin-2 in subjects with moderate-to-severe ulcerative colitis.
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 ...
Given the widespread use of anticonvulsants in the pediatric chronic pain population and the absence of scientific data supporting their use, the investigators propose a randomized, double blind, two ...
The purpose of the study is to 1) define the operating characteristics of fungal biomarker assays in pediatric patients at high-risk for developing invasive candidiasis, 2) determine the change in ...
This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical ...
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other ...
This is a randomized trial designed to evaluate testicular outcomes after a single stage versus a two-stage surgical procedure to fix an undescended, one-sided testicle located in the abdomen. Also to ...