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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Study Investigates Needs of Amyloidosis and Immunoglobulin Deposition Disease Patients During COVID-19
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Study Investigates Needs of Amyloidosis and Immunoglobulin Deposition Disease Patients During COVID-19

Throughout the COVID-19 pandemic, researchers have been dedicated to understanding which individuals are most at risk for severe disease. They've found that those who are immunocompromised and have certain cancers…

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What is the Recommended CAEL-101 Dose for AL Amyloidosis?
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What is the Recommended CAEL-101 Dose for AL Amyloidosis?

In early December 2020, many descended upon the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition to discuss insights in the field of hematology. During the Meeting, researchers…

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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…

Continue Reading A Potential Treatment for hATTR Amyloidosis has Earned Fast Track Designation
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…

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