RNA Interference

Latest News
February 25th 2021

Market of siRNAs Poised to Expand Beyond 3 Currently Approved Drugs

February 19th 2021

Early Diagnosis May Lead to More Mild Disease Course for Patients With AHP

February 18th 2021

Researchers Highlight Clinical Features of AHP, Emergence of siRNA-Based Therapies

January 29th 2021

Machine Learning May Help Diagnose Acute Hepatic Porphyria

January 28th 2021

Patients With Acute Hepatic Porphyria at Higher Risk of Accessing Long-term Sick Leave, Disability Pension

More News

Review Highlights Diagnostic Approaches, Treatment for ATTR
January 23rd 2021

Review Highlights Diagnostic Approaches, Treatment for ATTR

Gianna Melillo
In 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.
Women with gastrointestinal discomfort
January 20th 2021

Gastrointestinal Issues Prominent Among Patients With Hereditary ATTRv Amyloidosis

Matthew Gavidia
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

Lumasiran Approved for Patients With Primary Hyperoxaluria Type 1

AJMC Staff
Lumasiran works by inhibiting the production of oxalate.
Hands holding up liver
January 13th 2021

Givosiran Use Linked to Improvement in Porphyria Attacks

Maggie L. Shaw
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

Study Identifies Methylation Sites Linked to hATTR

Gianna Melillo
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

Patients, Caregivers Report Burden of Living With Primary Hyperoxaluria

Christina Mattina
Lumasiran Meets Primary End Point in Phase 3 Study in Patients With PH1
December 25th 2019

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

Kelly Davio
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

New Treatment Approved for Rare Genetic Disorder, Acute Hepatic Porphyria

Laura Joszt, MA
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

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

Laura Joszt, MA
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.
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June 13th 2017

Hereditary ATTR Amyloidosis: Burden of Illness and Diagnostic Challenges

Morie A. Gertz, MD, MACP

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