RNA Interference

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

December 25th 2019, 6:00pm

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

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

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