Showing results (21-30 of 39) with videos related to
Sort By:
Pageof 4
JACC. Advances|August 23, 2025
Vutrisiran in Transthyretin Amyloidosis: A Pooled Safety Analysis of HELIOS-A and HELIOS-BRonald M Witteles, Pablo Garcia-Pavia, Caroline Morbach, et al.Journal of Hepatology|July 21, 2023
Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trialDavid J Kuter, Herbert L Bonkovsky, Susana Monroy, et al.Pediatric Nephrology (Berlin, Germany)|August 1, 2022
Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trialWesley Hayes, David J Sas, Daniella Magen, et al.Neurology and Therapy|December 15, 2015
Delayed-Release Dimethyl Fumarate and Pregnancy: Preclinical Studies and Pregnancy Outcomes from Clinical Trials and Postmarketing ExperienceRalf Gold, J Theodore Phillips, Eva Havrdova, et al.Orphanet Journal of Rare Diseases|July 10, 2020
A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosisTeresa Coelho, David Adams, Isabel Conceição, et al.JAMA Cardiology|March 17, 2019
Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO StudyMasatoshi Minamisawa, Brian Claggett, David Adams, et al.Cardiovascular Drugs and Therapy|February 17, 2020
Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)Daniel P Judge, Arnt V Kristen, Martha Grogan, et al.Cardiovascular Drugs and Therapy|June 18, 2020
Correction to: Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)Daniel P Judge, Arnt V Kristen, Martha Grogan, et al.Liver International : Official Journal of the International Association for the Study of the Liver|October 30, 2021
Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION studyPaolo Ventura, Herbert L Bonkovsky, Laurent Gouya, et al.Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis|March 6, 2020
Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosisLaura Obici, John L Berk, Alejandra González-Duarte, et al.Pageof 4