Scientific Articles

Jan 10, 2021
Venetoclax Induces Deep Hematologic Remissions in t(11;14) Relapsed/Refractory AL Amyloidosis
Venetoclax is efficacious in relapsed/refractory t(11;14) multiple myeloma, thus warranting investigation in light-chain amyloidosis (AL). This retrospective cohort…
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Jan 10, 2021
The Patient Journey Toward a Diagnosis of Hereditary Transthyretin (ATTRv) Amyloidosis
Background: Despite emerging treatments for hereditary transthyretin (ATTRv) amyloidosis, the disease is often misdiagnosed, with reported diagnostic delays of up…
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Jan 06, 2021
Patient-Reported Burden of Hereditary Transthyretin Amyloidosis on Functioning and Well-Being
Hereditary transthyretin (hATTR) amyloidosis is a rare, systemic, progressive, and life-threatening disease in which transthyretin proteins misfold and aggregate as…
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Sep 22, 2020
Avoiding Misdiagnosis: Expert Consensus Recommendations for the Suspicion and Diagnosis of Transthyretin Amyloidosis for the General Practitioner
Background: Transthyretin amyloidosis (also known as ATTR amyloidosis) is a systemic, life-threatening disease characterized by transthyretin (TTR) fibril…
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Aug 06, 2020
CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future
A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across…
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Apr 30, 2020
Amyloid Cardiomyopathy
Cardiac amyloidosis is a heterogeneous group of diseases characterized by extracellular deposition of amyloid fibrils in many different organs finally resulting in…
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