RNA Interference

Market of siRNAs Poised to Expand Beyond 3 Currently Approved Drugs

February 25th 2021, 11:45pm

Article

While only 3 small interfering RNA (siRNA)-based therapies have been approved, the market is poised to expand with 7 other candidates in phase 3 trials.

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

February 19th 2021, 6:25pm

Article

Based on the findings, the researchers signal that family screening following a sentinel case may reap benefits for patients with acute hepatic porphyria (AHP).

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

February 18th 2021, 5:49pm

Article

A group of researchers discuss the clinical features and diagnosis of acute hepatic porphyrias (AHP) as well as their take on the approval of the first small interfering RNA (siRNA)-based therapy for the treatment of these patients.

Machine Learning May Help Diagnose Acute Hepatic Porphyria

January 29th 2021, 6:53pm

Article

Study results published in PLoS One strongly suggest machine learning can be an effective approach to identify patients who should receive a diagnostic biochemical test to screen for acute hepatic porphyria.

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

January 28th 2021, 6:30pm

Article

Patients with symptomatic acute hepatic porphyria were found to be at greater risk of accessing long-term sick leave and disability pension compared with the general population, but were not shown to be at an increased overall risk of premature death.

Review Highlights Diagnostic Approaches, Treatment for ATTR

January 23rd 2021, 5:30pm

Article

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.

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.