This is a pilot study aiming to examine preliminary effectiveness of a preparation program which includes simulation (SIMDiscovery) in reducing anxiety and increasing feelings of preparedness among ...
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative ...
ATHN 9 is a natural history study to assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric ...
This is an open-label, multicenter, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with mitapivat in participants who were previously enrolled in Study AG348 ...
The purpose of this study is to obtain preliminary data in advance of a larger clinical trial aimed to test whether a single session of green light exposure can lead to a clinically significant ...
The ICONICC Study is a randomized controlled clinical trial designed to compare the proportion of successful treatment outcomes between children assigned to standard concussion care only, standard ...
This is a Phase III clinical trial to compare the efficacy of two dosages of a new infant rehabilitation protocol - I-ACQUIRE - to usual and customary forms of infant rehabilitation in infants who ...
Effective patient education improves health literacy and engagement thus improving long-term health outcomes. Health literacy is imperative to make informed health decisions and relies on the ability ...
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).