115 recruiting studies matching your filters
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomar…
Arrhythmogenic ventricular cardiomyopathy (AVC) is a genetic condition which affects the heart and can lead to heart failure and rhythm problems, of which, sud…
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic di…
This study is a multi-functional integrated research and education project to prevent losses in academic achievement, cognitive function, and behavioral health…
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnos…
The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2…
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to neonatal encephal…
The current protocol is to determine the biodistribution, metabolism, excretion and brain uptake of 11C HY-2-15. The goal of this radiotracer is to quantify al…
This study is being done to further develop a device, the mScan, to measure muscle health as compared to measurements of muscle health using MRI (magnetic reso…
In this study, we will conduct retrospective chart and imaging reviews and prospective longitudinal virtual assessments of individuals with LBSL.
The purpose of this study is to systematically evaluate the results of medical investigations to identify symptom and biological patterns and common etiologies…