Transthyretin Amyloidosis: CRISPR-Cas9 Technology Shows Results in a Single Treatment

In August of 2021, Intellia Therapeutics of Cambridge and its partner Regeneron Pharmaceuticals published a study reported in the Boston Globe, showing results of a single treatment using CRISPR-Cas9 technology.…

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CRISPR Continues to Cut Impressive Numbers in Beta-Thalassemia, Sickle Cell Disease, and More

A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…

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AstraZeneca Enters Two Agreements for Transthyretin Amyloid Cardiomyopathy and Transthyretin Amyloidosis
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AstraZeneca Enters Two Agreements for Transthyretin Amyloid Cardiomyopathy and Transthyretin Amyloidosis

AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…

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Some Heart Failure Patients May Have an Underlying Condition Called Cardiac Amyloidosis

A recent article in the Northern Kentucky Tribune highlights a rare condition that has been exposed by new technology. Cardiologists have found that many patients diagnosed with heart failure are…

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Phase 3 International Trial for Transthyretin Amyloid Cardiomyopathy is Fully Enrolled
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Phase 3 International Trial for Transthyretin Amyloid Cardiomyopathy is Fully Enrolled

Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…

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Results from Phase 1 HATTR Amyloidosis Trial Presented at Recent Meeting
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Results from Phase 1 HATTR Amyloidosis Trial Presented at Recent Meeting

According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…

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You Can Help Raise Money for Amyloidosis Research This October
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You Can Help Raise Money for Amyloidosis Research This October

PW Partner the Amyloidosis Foundation (AF) is hosting their annual amyloidosis research benefit and you won't want to miss it! The AF was founded by two amazing amyloidosis patients who strived…

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