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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New Machine-Learning Model Shows Individuals If They’re at Risk of Rare Cardiomyopathy

According to a recent article, investigators created a machine learning model in order to identify if patients are at risk of rare cardiomyopathy. Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin amyloid cardiomyopathy…

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Measuring the Efficacy of Tafamidis as a Treatment for ATTR-CM
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Measuring the Efficacy of Tafamidis as a Treatment for ATTR-CM

When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…

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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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