Portuguese Authorities Approve Treatment for HATTR Amyloidosis Polyneuropathy
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Portuguese Authorities Approve Treatment for HATTR Amyloidosis Polyneuropathy

According to a story from PR Newswire, Akcea Therapeutics, Inc. recently announced that the Portuguese Medicine Regulatory Authority (INFARMED) has announced its approval of reimbursement for inotersen (marketed as TEGSEDI®)…

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Treatment for HATTR Amyloidosis Polyneuropathy Approved for Reimbursement in Spain
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Treatment for HATTR Amyloidosis Polyneuropathy Approved for Reimbursement in Spain

According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…

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A Potential Treatment for hATTR Amyloidosis has Earned Fast Track Designation
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A Potential Treatment for hATTR Amyloidosis has Earned Fast Track Designation

According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…

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Exclusive Distribution Agreement in Turkey for ONPATTRO to Treat Hereditary ATTR Amyloidosis
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Exclusive Distribution Agreement in Turkey for ONPATTRO to Treat Hereditary ATTR Amyloidosis

  According to a press release from FirstWord Pharma, Gen and Alnylam Pharmaceuticals have signed an exclusive distribution deal in Turkey for RNAi therapeutic ONPATTRO. This therapy drug will treat…

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First Treatment for Transthyretin Amyloidosis Cardiomyopathy Approved in Europe
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First Treatment for Transthyretin Amyloidosis Cardiomyopathy Approved in Europe

As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…

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VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy Received Positive Opinion by the (EMA) 

  Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the  European Medicines Agency’s (EMA) Committee for…

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Treating Colchicine-Resistant Familial Mediterranean Fever : Alternative Medications Can Succeed
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Treating Colchicine-Resistant Familial Mediterranean Fever : Alternative Medications Can Succeed

In an article from the Orphanet Journal of Rare Diseases, a recent study provides an overview of the treatment of familial Mediterranean fever (FMF) that does not respond to the standard…

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Companies Reach Partnership Agreement Related to an Experimental Treatment for ATTR Amyloidosis
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Companies Reach Partnership Agreement Related to an Experimental Treatment for ATTR Amyloidosis

According to a story from BioPortfolio, Alexion Pharmaceuticals, Inc. and Eidos Therapeutics, Inc., have recently struck an agreement related to the development and marketing of an experimental treatment for hATTR…

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New hATTR Amyloidosis Research Presented at Recent EU Meeting
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New hATTR Amyloidosis Research Presented at Recent EU Meeting

According to a story from BioPortfolio, the drug company Alnylam Pharmaceuticals recently presented research findings related to hATTR amyloidosis, a rare disease. The study was presented at the Second European…

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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 Approved for Transthyretin Amyloid Cardiomyopathy Will Cost $225,000 per Year

The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…

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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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