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Treatment of Dialysis-Related Amyloidosis Using Lixelle® β2-microglobulin Apheresis Column
Dialysis-related amyloidosis (DRA) is a serious complication of long-term hemodialysis (HD). Its pathogenic mechanism involves accumulation of β2-microglobulin…
Molecular Imaging of Primary Amyloid Cardiomyopathy
Cardiac amyloidosis is a major cause of early treatment-related death and poor overall survival in individuals with systemic light chain amyloidosis. This proj…
A Randomized, Participant & Investigator Blinded, Placebo-Controlled Study to Evaluate the Ability of Intrathecally Administered NIO752 to Lower CSF Total Tau Synthesis in Participants With AD Measured by Stable Isotope Labelling Kinetics
This study will assess if drug (NIO752) reduces production of a protein, tau, by the brain. Normally tau maintains the internal skeleton of nerve cells. In Alz…
Myocardial & Hepatic Shear Wave Velocity Measurements in Patients With Non-Reduced-Ejection Fraction Heart Failure and Control Subjects - US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failu…
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
An Open-label, Multi-center, Non-randomized Pivotal Phase 3 Study to Evaluate the Efficacy and Safety of [18F]Florbetaben Positron Emission Tomography (PET) Imaging to Diagnose Cardiac AL Amyloidosis
This is an open-label, multi-center pivotal Phase 3 study to visually and quantitatively assess PET images obtained after single application of 300 MBq \[18F\]…
Minimal Residual Disease Response-adapted Deferral of Transplant in Dysproteinemia - MILESTONE Trial
This is a phase II interventional study evaluating the use of minimal residual disease by next generation sequencing to defer autologous hematopoietic stem cel…
Myocardial Stiffness in Amyloidosis by Magnetic Resonance Elastography - AMREloid Study
This is a prospective case control study to determine the feasibility and utility of myocardial stiffness assessment by cardiac magnetic resonance elastography…