‘Anyone with a Heart’ Campaign Spreads Awareness About Hereditary ATTR Amyloidosis
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‘Anyone with a Heart’ Campaign Spreads Awareness About Hereditary ATTR Amyloidosis

Fredericksburg, Virginia resident Johnny Boatman is a former national guardsman who had been in excellent physical condition all his life. That was until 2022 when he felt a sharp pain…

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FDA Denies Approval of Onpattro for Cardiomyopathy of ATTR Amyloidosis
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FDA Denies Approval of Onpattro for Cardiomyopathy of ATTR Amyloidosis

  In 2018, the U.S. Food and Drug Administration (FDA) approved Onpattro (patisiran) as a treatment for polyneuropathy in people with hereditary transthyretin-mediated (ATTR) amyloidosis. Onpattro is an intravenously administered,…

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Transthyretin Amyloid Cardiomyopathy: An Underrecognized Disease in the Black Community
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Transthyretin Amyloid Cardiomyopathy: An Underrecognized Disease in the Black Community

  The latest statistics on heart disease and its effect on Black communities are startling. According to an article in the Dallas Examiner, Black people are fifty to seventy percent more…

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UC San Diego Health First in US to Offer Amvuttra for ATTR Amyloidosis
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UC San Diego Health First in US to Offer Amvuttra for ATTR Amyloidosis

  In June 2022, the FDA approved Amvuttra, a prescription medicine designed to treat polyneuropathy (nerve damage) due to hereditary transthyretin amyloidosis (hATTR amyloidosis). This approval was huge. Typically, managing…

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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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