RNA Interference

Gastrointestinal Issues Prominent Among Patients With Hereditary ATTRv Amyloidosis

January 20th 2021, 10:30pm

Article

A 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.

Lumasiran Approved for Patients With Primary Hyperoxaluria Type 1

January 16th 2021, 10:45am

Article

Lumasiran works by inhibiting the production of oxalate.

Givosiran Use Linked to Improvement in Porphyria Attacks

January 13th 2021, 7:47pm

Article

Use 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.

Study Identifies Methylation Sites Linked to hATTR

January 8th 2021, 2:30pm

Article

Researchers 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.

Patients, Caregivers Report Burden of Living With Primary Hyperoxaluria

January 3rd 2021, 1:30pm

Article

Lumasiran Meets Primary End Point in Phase 3 Study in Patients With PH1

December 25th 2019, 6:00pm

Article

Drug 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).

New Treatment Approved for Rare Genetic Disorder, Acute Hepatic Porphyria

November 21st 2019, 3:23am

Article

The 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.

Study Finds Unmet Need for Therapies for Recurrent Attacks of Acute Hepatic Porphyria

October 20th 2019, 6:00pm

Article

Data 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.

Hereditary ATTR Amyloidosis: Burden of Illness and Diagnostic Challenges

June 13th 2017, 12:46pm

Article