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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 \[18F\]…
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like sub…
A Phase 1/2 Study of Linvoseltamab in Patients With Relapsed or Refractory Systemic Light Chain Amyloidosis
This study is researching an experimental drug called linvoseltamab ("study drug"). This study is focused on patients who have AL amyloidosis that has returne…
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…