This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll ...
This 2-year study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic (PD) effects of ocrelizumab in children and adolescents ages ≥ 10 to ≤ 18 years with relapsing-remitting ...
This pilot study aims to assess the feasibility, acceptability, and effectiveness of using nitrous oxide (N2O) sedation for intrauterine device (IUD) insertions for nulliparous adolescent and young ...
The objectives of this multicenter pragmatic clinical trial are to compare the effectiveness and relative safety of balanced fluid resuscitation versus 0.9% "normal" saline in children with septic ...
In this research study we want to learn more about the character of neutrophils that are present in the blood of children with sepsis. Sepsis is a severe type of infection, affecting various parts of ...
In this research study we want to learn more about the characteristics of neutrophils that are present in the blood and secretions from a breathing tube of patients with sepsis. Sepsis is a severe ...
Investigators want to learn the role of indoor environmental exposures on respiratory symptoms, and, separately, on lung function deficits in school-aged children with bronchopulmonary dysplasia (BPD) ...
Pain remains a common and frequently debilitating symptom, particularly during adulthood of Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS). For many FD/MAS patients, the amount of pain perceived ...
This research study is designed to study the combination of two drugs, palbociclib and ganitumab, as a potential treatment for Ewing sarcoma. The names of the study drugs involved in this study are: ...
The primary objective of this study is to evaluate several interventions given to participants with severe asthma. Interventions are administered in a crossover manner with 16-week treatment periods ...