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Report From the Amyloidosis Forum Scientific Workshop
The Amyloidosis Forum, a Public Private Partnership with ARC and FDA, hosted the workshop on 22nd January, as part of an ongoing process to develop novel endpoints for clinical trial in AL amyloidosis. In 2020, the Amyloidosis Forum launched a series of meetings focused on Novel Endpoints and Analyses for Clinical Trials in AL amyloidosis. Following the first meeting held in October 2020, specialized working groups in the areas of cardiac, hematological, renal, and other organ systems were formed. Additional...
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Treatment Overview: Hereditary Transthyretin Amyloidosis
We live in a time of rapid advances in genetic know-how and pharmacological technologies. The pace of discovery is accelerating, driving the development of new therapies, with 3 newly approved treatments. The exact course of hATTR varies with each patient, but the outlook holds promise for all. This booklet is designed as a comprehensive guide to help you and your family navigate treatment resources and options that would be most effective for you.
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Disease Overview: Hereditary Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis (hATTR) is a rare, systemic disease passed down through families. Caused by genetic mutations in the transthyretin (TTR) gene, it leads to a buildup of abnormal proteins called amyloid in one or more organs and tissues, impairing their function. Left untreated, hATTR can cause life-threatening complications. Early diagnosis and treatment are critical to prevent or delay progression of hATTR.
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Disease and Treatment Overview: Wild-Type Transthyretin Amyloidosis
Wild-type transthyretin amyloidosis (ATTRwt) is an age-related disease caused by transthyretin (TTR) proteins that become unstable, misfold, and build up in organs, impairing their function. It is a slowly progressive condition that affects older people, most often Caucasian men over 65 years of age. Heart disease is the hallmark of ATTRwt, but it is commonly preceded by other conditions, such as carpal tunnel syndrome or spinal stenosis.
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About Amyloidosis
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