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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 janeiroEstado do rioUnited statesNew zealandCidade universitariaAlnylam pharmacauticalsMarques jrClementino fraga filhoWaddington cruzIonis astrazenecaRodolpho paulo roccoIntellia regeneronResearch to practiceAmerican academy of neurology annual meetingIonis pharmaceuticalsIonis pharmaceuticals inc

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 statesRichard smithJuliand gillmoreByerik swainIntellia regeneronCentre for amyloidosisAmerican heart association scientific sessionsPhase proteins at university college londonNovo nordiskBreaking scienceBreakthrough strategiesAmerican heart association scientificAcute phase proteinsUniversity 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 statesMarianna fontanaAlison sonderfanLiron walshDavide gutsteinYuanxin xuTed ganeAdam boydJeffrey cehelskyMichael maitlandMark strohJuliand gillmoreDavid lebwohlIntellia regeneronAmerican heart associationUniversity college london centre for amyloidosis

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 regeneronEric topolJohn leonardRegeneron pharmaceuticalsScripps research translational instituteRegeneron pharmaceuticals incIntellia therapeutics incNobel prize winningIntellia therapeuticsNew england journalஎரிக் தோப்போல்ஜான் லியோனார்ட்ஸ்கிரிப்ட்கள் ஆராய்ச்சி மொழிபெயர்ப்பு நிறுவனம்நோபல் ப்ரைஸ் வென்றதுபுதியது இங்கிலாந்து இதழ்