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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: Light Chain (AL) Amyloidosis
AL amyloidosis is a serious disease. However, many patients benefit from current therapies, with their lives improved and prolonged, often for many years.
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Disease Overview: Light Chain (AL) Amyloidosis
A diagnosis of light chain amyloidosis (AL amyloidosis) can be confusing and stressful, bringing up many feelings and questions. It is important to learn as much as possible about the disease, its treatment, and how it might affect you.
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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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