Brineura is recombinant human tripeptidyl peptidase 1 (TPP1) replacement enzyme to slow loss of walking ability in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is infused into the cerebrospinal fluid (CSF) via an intraventricular access device implanted in the patient’s head. It is administere…
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.
Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Systemic Sclerosis
Health Problem: Systemic sclerosis (SSc) is a progressively disabling autoimmune disease of the connective tissue that causes skin thickening; the disease can thicken visceral connective tissue that results in organ impairment, organ failure, and mortality. SSc is a rare disease, estimated to affect 250 people per 1,000,000 population in the …
Onpattro (Patisiran)
Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to …
Onpattro (Patisiran)
Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to …
Hybrid Maze Procedure for Atrial Fibrillation
Health Problem: Atrial fibrillation (AF) is a supraventricular, accelerated heart rhythm characterized by uncoordinated atrial activation that leads to inefficient, irregular atrial contraction. According to 2010 data, approximately 2.7 to 6.1 million people in the United States have AF. In both men and women, AF is associated with a signific…
Ultomiris (Ravulizumab-cwvz) for Paroxysmal Nocturnal Hemoglobinuria
Ultomiris is a long-acting humanized murine monoclonal antibody that is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ultomiris binds with high affinity to the complement protein C5, thereby inhibiting its cleavage of C5a and C5b. This inhibition prevents the generation of the terminal complement complex C5b9, preventing complement-mediated hemolys…
Ultomiris (Ravulizumab-cwvz) for Paroxysmal Nocturnal Hemoglobinuria
Ultomiris is a long-acting humanized murine monoclonal antibody that is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ultomiris binds with high affinity to the complement protein C5, thereby inhibiting its cleavage of C5a and C5b. This inhibition prevents the generation of the terminal complement complex C5b9, preventing complement-mediated hemolys…
Luxturna (Voretigene Neparvovec-rzyl) for Inherited Retinal Dystrophies
Luxturna (voretigene neparvovec-rzyl) is an adenovirus-associated viral vector serotype 2 gene therapy containing the human RPE65 gene for treatment of patients with vision loss due to RPE65-mediated inherited retinal disease. Gene therapy utilizes a viral vector to carry the desired genetic information to target c…
Luxturna (Voretigene Neparvovec-rzyl) for Inherited Retinal Dystrophies
Luxturna (voretigene neparvovec-rzyl) is an adenovirus-associated viral vector serotype 2 gene therapy containing the human RPE65 gene for treatment of patients with vision loss due to RPE65-mediated inherited retinal disease. Gene therapy utilizes a viral vector to carry the desired genetic information to target c…
Libtayo (Cemiplimab)
Libtayo (cemiplimab) is an intravenous programmed death receptor-1 (PD-1) blocking antibody indicated for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.