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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ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK
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ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK

  An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…

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Patisiran for HATTR Amyloidosis: How a Patient Got Involved
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Patisiran for HATTR Amyloidosis: How a Patient Got Involved

According to a story from Nature, Dr. Carlos Heras-Palou was diagnosed with hATTR amyloidosis when he was just 39 years old. Initially, the devastating diagnosis was completely overwhelming, as the disease…

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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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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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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…

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Worried About hATTR Amyloidosis? Check Out This New Genetic Testing Program
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Worried About hATTR Amyloidosis? Check Out This New Genetic Testing Program

According to Globe Newswire, a pharmaceutical company, Akcea Therapeutics, recently announced their new genetic testing and counseling treatment program for those who may have hereditary ATTR amyloidosis (hATTR amyloidosis). The program,…

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Investigational Treatment for hATTR Amyloidosis Shows Promising Results in Trials

According to a story from Rare Disease Report, the pharmaceutical company Alnylam Pharmaceuticals announced the results of Phase 3 trials for its experimental drug patisiran. The drug is in the…

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