Tag: Familial amyloid polyneuropathy

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Recent Related Articles to Familial amyloid polyneuropathy

CRISPR gene-editing therapy shows promise as cardiac amyloidosis treatment in early study

Added to Collection on: 2024-11-16 18:02:37

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Amyloidosis
Familial amyloid polyneuropathy
Familial amyloid cardiomyopathy
Alnylam Pharmaceuticals
Transthyretin
Cardiac amyloidosis
Heart failure
Email

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Intellia's CRISPR Therapy Shows 90%+ Disease Protein Reduction in Key Clinical Trial

Added to Collection on: 2024-11-17 02:14:16

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Amyloidosis
Cardiomyopathy
Neurological disorders
Protein folding
Familial amyloid polyneuropathy
Familial amyloid cardiomyopathy
Intellia Therapeutics
Transthyretin
Brain natriuretic peptide 32
Heart failure
Acoramidis
Wild-type transthyretin amyloid
CRISPR

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New gene editing therapy 'safe and effective' - Royal Free London NHS Foundation Trust

Added to Collection on: 2024-11-16 15:46:38

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Amyloidosis
Transthyretin
Intellia Therapeutics
Wild-type transthyretin amyloid
Therapy
Familial amyloid polyneuropathy

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Intellia Announces First Clinical Evidence from Ongoing - GlobeNewswire

Added to Collection on: 2024-11-16 15:31:31

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Amyloidosis
Neurological disorders
Cardiomyopathy
Protein folding
Familial amyloid polyneuropathy
Familial amyloid cardiomyopathy
Transthyretin
Intellia Therapeutics
Troponin
Vutrisiran
Wild-type transthyretin amyloid
CRISPR

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New gene editing therapy for heart condition is safe and effective | UCL News

Added to Collection on: 2024-11-19 22:11:36

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Amyloidosis
CRISPR gene editing
Familial amyloid polyneuropathy
Intellia Therapeutics
Transthyretin
Therapy
Wild-type transthyretin amyloid
Gene therapy

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Novel CRISPR-Cas9 Therapy Slows ATTR-CM Progression | tctmd.com

Added to Collection on: 2024-11-20 21:29:53

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Amyloidosis
Orphan drugs
Neurological disorders
Cardiomyopathy
Small interfering RNA
Vutrisiran
Familial amyloid polyneuropathy
Transthyretin
Familial amyloid cardiomyopathy
Alnylam Pharmaceuticals
Brain natriuretic peptide 32
Tafamidis

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Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT ...

Added to Collection on: 2024-11-25 17:33:34

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Amyloidosis
Neurological disorders
Pharmaceuticals policy
Intellia Therapeutics
Protein folding
Familial amyloid polyneuropathy
Transthyretin
Regenerative Medicine Advanced Therapy
Orphan drug
Wild-type transthyretin amyloid
Alnylam Pharmaceuticals
CRISPR
Intellia Therapeutics Inc

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Acoramidis Receives Positive CHMP Opinion for Treatment of Transthyretin ... - The Manila Times

Added to Collection on: 2024-12-13 18:50:25

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Cardiomyopathy
Amyloidosis
Fluoroarenes
Acoramidis
Pyrazoles
Familial amyloid polyneuropathy
Transthyretin
Familial amyloid cardiomyopathy
Bayer
Clinical trial
European Medicines Agency
Forward-looking statement
United States

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CGTLive®'s Weekly Rewind – March 21, 2025

Added to Collection on: 2025-03-21 19:08:22

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Biotechnology
Applied genetics
Muscular dystrophy
Rare diseases
Bioethics
Gene therapy
Duchenne muscular dystrophy
Delandistrogene moxeparvovec
Familial amyloid polyneuropathy
Therapy
Sarepta Therapeutics
Toshifumi Yokota

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Intellia Therapeutics' Nex-z Receives FDA Regenerative Medicine Advanced Therapy Designation

Added to Collection on: 2025-03-26 13:42:53

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Amyloidosis
Neurological disorders
Orphan drugs
Familial amyloid polyneuropathy
Transthyretin
Intellia Therapeutics
Vutrisiran
u.s. food and drug administration
Intellia Therapeutics Inc

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