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Top Rare Disease Advisor News

Acoramidis Snags FDA Approval for ATTR Cardiomyopathy
United States · United States

Acoramidis Snags FDA Approval for ATTR Cardiomyopathy

100% Center coverage: 1 sources
(MedPage Today) -- Acoramidis (Attruby) gained FDA approval for treating wild-type or variant transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM), BridgeBio Pharma announced. Offering patients near-complete TTR stabilization in an oral tablet...See the Story
Israeli Study Could Lead to Gene Therapy for Cancer, Brain Diseases
Cancer

Israeli Study Could Lead to Gene Therapy for Cancer, Brain Diseases

100% Right coverage: 1 sources
"This study provides new, groundbreaking insights in genetic medicine, which may lead to the development of innovative treatments for severe diseases.”See the Story
'Designer Cells' Could Be the Key to Autoimmune Disease Treatment

'Designer Cells' Could Be the Key to Autoimmune Disease Treatment

100% Left coverage: 1 sources
Scientists are beginning to explore the use of designer cells in autoimmune disease treatment. According to the National Cancer Institute, an autoimmune disease is “a condition in which the body’s immune system mistakes its own healthy tissues as foreign and attacks them.” These diseases often lead to high inflammation within the body, as well as symptoms like fevers, fatigue, aches and pains, swelling, skin rashes, digestive problems, and more.…See the Story

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