This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA). The study consists of 3 parts ...
Surgical site infections (SSIs) are one of the major hospital acquired infections and responsible for the most cost among the hospital acquired infections. The objective of this study is to assess the ...
To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome
The goal of this research study is to to learn more about the body's immune response in patients with multiple sclerosis (MS). In MS, the body's immune cells mistakenly attack an important part of the ...
This is a prospective observational study to investigate if a non-invasive blood pressure monitoring device, the CareTaker Pulse Decomposition Analysis (PDA), can accurately measure blood pressure in ...
Background: Current pain management strategies for pediatric patients are not integrating the analgesic potential of movement-based therapies. To date, experiencing a painful stimulus has been known ...
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure. The study will ...
In this study, the investigators aim to evaluate changes in lung function in women with cystic fibrosis (CF) during pregnancy and for 2 years after pregnancy based on exposure to highly effective ...
This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec ...