This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3704 in patients with methylmalonic acidemia/aciduria (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency between 1 to 18 years ...
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment ...
The purpose of this study is to test whether an external erectile prosthesis (The Elator™) is a feasible alternative to internal erectile prostheses for transgender men who have undergone a ...
In this research study the investigators want to learn more about how a medication called tranexamic acid (TXA) could help reduce bleeding during Periacetabular Osteotomy (PAO) surgery. TXA is ...
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 ...