Review Highlights Diagnostic Approaches, Treatment for ATTR
January 23rd 2021In a review published in Therapeutics and Clinical Risk Management, researchers outlined the current understanding of the transthyretin amyloidosis (ATTR) disease process, diagnostic and surveillance approaches, treatment modalities, and potential future directions.
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Gastrointestinal Issues Prominent Among Patients With Hereditary ATTRv Amyloidosis
January 20th 2021A stark 82% of assessed patients with hereditary transthyretin (ATTRv) amyloidosis exhibited at least 1 gastrointestinal symptom, warranting the need for gastroenterologists in multidisciplinary management of the disease.
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Givosiran Use Linked to Improvement in Porphyria Attacks
January 13th 2021Use of the RNA inhibitor givosiran was shown to reduce expression of delta-aminolevulinic acid synthase 1, in turn reducing the severity of acute attacks and chronic symptoms for patients with severe hepatic porphyria.
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Study Identifies Methylation Sites Linked to hATTR
January 8th 2021Researchers conducting a large epigenome-wide association study identified several methylation sites associated with hereditary transthyretin amyloidosis (hATTR), a rare, life-threatening disorder caused by amyloidogenic coding mutations in the TTR gene.
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Lumasiran Meets Primary End Point in Phase 3 Study in Patients With PH1
December 25th 2019Drug maker Alnylam Pharmaceuticals has announced that the ILLUMINATE-A trial, a phase 3 study of lumasiran, an investigational RNA interference product targeting glycolate oxidase, met its primary end point in a study of patients with primary hyperoxaluria type 1 (PH1).
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New Treatment Approved for Rare Genetic Disorder, Acute Hepatic Porphyria
November 21st 2019The FDA has approved a new treatment for adult patients with acute hepatic porphyria (AHP) a rare genetic disorder. Givlaari is an RNA interference therapeutic targeting aminolevulinic acid synthase 1. Simultaneously, Alnylam Pharmaceuticals announced a new framework for value-based agreements to help patients gain access to the treatment.
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Study Finds Unmet Need for Therapies for Recurrent Attacks of Acute Hepatic Porphyria
October 20th 2019Data from EXPLORE, a prospective, multinational, natural history study, is used to characterize disease activity and clinical management of patients with acute hepatic porphyria (AHP) who experience recurrent attacks. The findings highlight the high unmet need for effective treatments.
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