February 25th 2021, 11:45pm
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.
February 19th 2021, 6:25pm
Based on the findings, the researchers signal that family screening following a sentinel case may reap benefits for patients with acute hepatic porphyria (AHP).
February 18th 2021, 5:49pm
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.
January 29th 2021, 6:53pm
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.
January 28th 2021, 6:30pm
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.
January 23rd 2021, 5:30pm
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.
January 20th 2021, 10:30pm
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.
January 16th 2021, 10:45am
Lumasiran works by inhibiting the production of oxalate.
January 13th 2021, 7:47pm
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.
January 8th 2021, 2:30pm
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.
