After Diagnosis, This Man Traced the Origins of His Family’s hATTR Amyloidosis
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After Diagnosis, This Man Traced the Origins of His Family’s hATTR Amyloidosis

According to a story from Wapakoneta Daily News, Greg was devastated when he learned that his brother was diagnosed with hereditary transthyretin mediated (hATTR) amyloidosis. Ultimately, the disease would take…

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Transthyretin-Mediated Amyloidosis (ATTR-CM); After Nine Years Pfizer Wins FDA Breakthrough Designation

It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…

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First Ever Treatment for Transthyretin-Mediated Amyloidosis Cardiomyopathy Approved by FDA

Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM).  ATTR-CM is a rare disease that occurs when amyloids, an…

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New Treatment for Familial Chylomicronemia Syndrome is Close to Approval in the EU

Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…

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BridgeBio Receives Almost 300 Million for Continued Rare Disease Research

About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…

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A Recap on 2018: Record Number of Drugs Approved by the FDA for Rare Diseases

Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…

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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development
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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development

According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…

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Company Plans Clinical Trial of Autonomic Regulation Therapy to Treat Heart Failure
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Company Plans Clinical Trial of Autonomic Regulation Therapy to Treat Heart Failure

According to a story from Compelo, the medical technology company LivaNova is kicking off an international trial that will assess the use of autonomic regulation therapy as a treatment for…

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Risk of Heart Failure in Older Women Can be Reduced With Exercise, Study Says
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Risk of Heart Failure in Older Women Can be Reduced With Exercise, Study Says

According to a story from Reuters, a recent study found that mild exercise, such as walking, can significantly reduce the risk of heart failure in older, postmenopausal women. This large…

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This Surgeon Preserved His Hand Function Thanks to New Amyloidosis Drug
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This Surgeon Preserved His Hand Function Thanks to New Amyloidosis Drug

According to a story from the Irish Examiner, treatment with the recently approved drug patisiran helped a British surgeon named Carlos Heras-Palou save both his hands and his career. Patisiran…

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Research Shows a Drug For Transthyretin Amyloid Cardiomyopathy May Significantly Reduce Mortality in Patients

  Pfizer has released the primary results from the Phase 3 clinical trial of tafamidis, an investigational drug being researched as a potential treatment for transthyretin amyloid cardiomyopathy. For more…

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The FDA Has Granted Orphan Drug Status to an Experimental Drug to Treat Amyloidosis

The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…

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Is There a Genetic Condition in Your Family?
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Is There a Genetic Condition in Your Family?

Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…

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New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis
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New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis

According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…

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The Results of a Pivotal Study of Tegsedi For The Treatment of hATTR Amyloidosis With Polyneuropathy Have Been Published
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The Results of a Pivotal Study of Tegsedi For The Treatment of hATTR Amyloidosis With Polyneuropathy Have Been Published

The final results from a study of the experimental drug tegsediTM (inotersen) for the treatment of patients with hereditary ATTR amyloidosis with polyneuropathy have been published. The original article can be…

Continue Reading The Results of a Pivotal Study of Tegsedi For The Treatment of hATTR Amyloidosis With Polyneuropathy Have Been Published