Results 61 to 70 of about 2,204 (179)

An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

Journal of Market Access & Health Policy, 2021
Background: Gene therapies can treat, prevent, or cure a disease by changing the expression of a person’s genes. They are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare ...
Michał Pochopień, Ewelina Paterak, Emilie Clay, Justyna Janik, Samuel Aballea, Małgorzata Biernikiewicz, Mondher Toumi   +6 more
doaj   +1 more source

Autism spectrum disorder in children with spinal muscular atrophy type 1: Case series

Developmental Medicine &Child Neurology, EarlyView.
Autism spectrum disorder (ASD) was identified in 37.5% of children with SMA (n = 13). While IQ did not differ significantly between groups, adaptive functioning was reduced in those with co‐occurring ASD. Results support routine early neurodevelopmental screening in children with spinal muscular atrophy.
Lorena V. Rezende, Adriana B. Ortega, Daniel A. Do Valle, Tiago S. Bara, Mara L. Cordeiro   +4 more
wiley   +1 more source

Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models [PDF]

, 2021
Spinal muscular atrophy (SMA) is a severe childhood neuromuscular disease for which two genetic therapies, Nusinersen (Spinraza, an antisense oligonucleotide), and AVXS-101 (Zolgensma, an adeno-associated viral vector of serotype 9 AAV9), have recently ...
Hirst, TC, Chilcott, EM, Yáñez-Muñoz, RJ, Muiruri, EW   +3 more
core   +1 more source

Comparison of Wildtype and Recombinant Adeno‐Associated Virus (AAV) Production Reveals Distinct Productivity and Content Ratio Profiles

Biotechnology Journal, Volume 21, Issue 11, November 2026.
Graphical Abstract and Lay Summary Wildtype and recombinant adeno‐associated virus production are compared across infection, transfection, and hybrid systems in suspension HEK293 cells. Wildtype infection achieves markedly higher vector genome titers and content ratios than triple transfection, and adenoviral helper input governs both packaging ...
Sofia Alfieri, Kelvin H. Lee
wiley   +1 more source

Cross‐Neutralizing and Broadly Neutralizing Human Antibodies Against Nonenveloped Viruses

European Journal of Immunology, Volume 56, Issue 10, October 2026.
In this review article, Marchand et al. compare cross ‐ and broadly ‐neutralizing human antibodies against non ‐ enveloped viruses from several major families, discussing their structural characteristics, neutralization mechanisms and relevance for the development of antiviral vaccines and therapeutics.
Sarah Marchand, Samy Taouli, Dorian McIlroy   +2 more
wiley   +1 more source

Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives

Therapeutics and Clinical Risk Management, 2019
Tamara Dangouloff,1 Laurent Servais1,2 1Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium ...
Dangouloff T, Servais L
doaj  

Engineering mRNA‐LNP Medicines for the Ageing Brain: Opportunities and Challenges for Neurodegenerative Diseases

Exploration, Volume 6, Issue 5, October 2026.
This review highlights recent advances in engineering messenger RNA (mRNA)‐lipid nanoparticles (LNPs) to cross the ageing blood–brain barrier and target neurodegenerative diseases. It outlines design principles, delivery routes, and translational challenges, charting a roadmap towards clinical application of mRNA‐LNP therapeutics for neurodegenerative ...
Abdel Ali Belaidi, Denzil L. Furtado, Francesca Alves, Rebecca M. Nisbet, Scott Ayton, Ashley I. Bush   +5 more
wiley   +1 more source

Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?

Neurotherapeutics
Adeno-associated virus (AAV) mediated gene therapy is a leading gene delivery platform with potential to transform the landscape of treatment for neurological disorders.
Ashley L. Harkins, Prajakta P. Ambegaokar, Allison M. Keeler   +2 more
doaj   +1 more source

Status and future of recombinant adeno‐associated virus vector manufacturing

Biotechnology Progress, Volume 42, Issue 5, September/October 2026.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

Fortalecimento de capacidades estatais: estudo de caso sobre a atuação dos NATJUS na incorporação do Zolgensma para o tratamento da Atrofia Muscular Espinhal Tipo 1 [PDF]

, 2023
Por meio deste estudo de caso da atuação dos Núcleos de Apoio Técnico ao Poder Judiciário – NATJUS, vinculados aos Tribunais de Justiça de São Paulo e do Distrito Federal e Territórios, lança-se luz às potencialidades advindas da aplicação dos marcos ...
Dias Júnior, Osmar Sebastião
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