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Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy

This open-label, single-group, phase 3 trial evaluates eplontersen, an investigational ligand-conjugated antisense oligonucleotide, in adults with hereditary am ....

Rio De Janeiro , Estado Do Rio , United States , New Zealand , Cidade Universitaria , Alnylam Pharmacauticals , Marques Jr , Clementino Fraga Filho , Waddington Cruz , Ionis Astrazeneca , Rodolpho Paulo Rocco , Intellia Regeneron , Research To Practice , American Academy Of Neurology Annual Meeting , Ionis Pharmaceuticals , Ionis Pharmaceuticals Inc , University Of Rio De Janeiro , Aptitude Health , Bristol Myers Squibb , Novo Nordisk , Group Information , Drug Administration , Alnylam Pharmaceuticals , Peripheral Nerve Society Annual Meeting , International Council For Harmonisation , Mayo Clinic Peripheral Nerve Research Laboratory ,

CRISPR-based genome editing therapy may help in ATTR amyloidosis with cardiomyopathy

CHICAGO — A novel CRISPR-based in vivo gene editing therapy reduced transthyretin levels in patients with hereditary transthyretin amyloidosis with cardiomyopathy, researchers reported at the American Heart Association Scientific Sessions. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a “progressive and fatal disease,” during which amyloid deposits cause impaired ....

United States , Richard Smith , Juliand Gillmore , Byerik Swain , Intellia Regeneron , Centre For Amyloidosis , American Heart Association Scientific Sessions , Phase Proteins At University College London , Novo Nordisk , Breaking Science , Breakthrough Strategies , American Heart Association Scientific , Acute Phase Proteins , University College London ,

Novel gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy

Novel gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy
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United States , Marianna Fontana , Alison Sonderfan , Liron Walsh , Davide Gutstein , Yuanxin Xu , Ted Gane , Adam Boyd , Jeffrey Cehelsky , Michael Maitland , Mark Stroh , Juliand Gillmore , David Lebwohl , Intellia Regeneron , American Heart Association , University College London Centre For Amyloidosis , American Heart Association Scientific Sessions , Research Highlights , Scientific Sessions , University College London Centre ,

Here's why Intellia Therapeutics stock gained 55% on Monday

Here's why Intellia Therapeutics stock gained 55% on Monday
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Intellia Regeneron , Eric Topol , John Leonard , Regeneron Pharmaceuticals , Scripps Research Translational Institute , Regeneron Pharmaceuticals Inc , Intellia Therapeutics Inc , Nobel Prize Winning , Intellia Therapeutics , New England Journal , எரிக் தோப்போல் , ஜான் லியோனார்ட் , ஸ்கிரிப்ட்கள் ஆராய்ச்சி மொழிபெயர்ப்பு நிறுவனம் , நோபல் ப்ரைஸ் வென்றது , புதியது இங்கிலாந்து இதழ் ,