Etranacogene dezaparvovec (Hemgenix; formerly AMT-061) is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Publications
Hayes Publications are available through the Hayes Knowledge Center, a portal to world-class research and unbiased analyses on the clinical, financial, and risk impacts of emerging, controversial, and disruptive healthcare technologies.Every Hayes publication is developed by multidisciplinary teams of clinicians and scientists using rigorous methods grounded in internally recognized standards for systematic review and Health Technology Assessment. Learn more about these evidence resources and see sample titles below.
See below for some of our recent publications.
Cologuard (Exact Sciences Corp.) for Colorectal Cancer Screening in Average-Risk Adults
This report evaluates the clinical validity and clinical utility of the Cologuard test (Exact Sciences Corp.).
Zavegepant (Zavzpret) for Acute Treatment of Migraine
Zavegepant is an intranasal novel small-molecule, calcitonin gene-related peptide (CGRP) receptor antagonist indicated for the acute treatment of migraine with or without aura in adults.
Omecamtiv Mecarbil for Heart Failure With Reduced Ejection Fraction (HFrEF)
Omecamtiv mecarbil is a first-in-class oral cardiac myosin activator under investigation for the treatment of heart failure with reduced ejection fraction (HFrEF).
Trofinetide (Daybue) for Rett Syndrome
Trofinetide (Daybue) is an oral synthetic glypromate analog indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
Repetitive Transcranial Magnetic Stimulation for Treatment of Bipolar Disorder
Focus of the Report: The focus of this Health Technology Assessment is to evaluate the effectiveness and safety of repetitive transcranial magnetic stimulation (rTMS) for the treatment of adult patients with bipolar disorder (BD).
Technology Description: Transcranial magnetic stimulati…
ThyroSeq v3 Genomic Classifier (University of Pittsburgh Medical Center and Sonic Healthcare USA)
This report evaluates the clinical validity and clinical utility of the ThyroSeq v3 Genomic Classifier (University of Pittsburgh Medical Center and Sonic Healthcare USA).
Omaveloxolone (Skyclarys) for Friedreich Ataxia
Omaveloxolone is an oral drug that activates nuclear factor erythroid 2-related factor 2 (Nrf2). It is intended for treatment of Friedreich ataxia (FA), a rare hereditary neurodegenerative disorder that is driven by mitochondrial dysfunction and impaired Nrf2 activity. FA typically presents between the ages of 10 and 15 years.
MyoPro Orthosis (Myomo Inc.) for Upper Extremity Paralysis/Paresis After Stroke
This report addresses whether full-text clinical studies, systematic reviews, and clinical practice guidelines and position statements support the use of the MyoPro orthosis (Myomo Inc.) for upper extremity paralysis/paresis after stroke to reduce motor impairment.
LiverMultiScan (Perspectum) for Diagnosis of Liver Fibrosis and Steatosis Related to Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis
Focus of the Report: This Health Technology Assessment focuses on evaluating the effectiveness and safety of the LiverMultiScan compared with other imaging and blood marker tests for assessment of liver fibrosis or steatosis in patients who have known or suspected non-alcoholic fatty liver disease (NAFLD) or non-alcoholic steatohepatitis (NAS…