Results 61 to 70 of about 2,159 (173)

Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment

Frontiers in Pharmacology, 2020
Spinal muscular atrophy (SMA) is the most common genetic disease affecting infants and young adults. Due to mutation/deletion of the survival motor neuron (SMN) gene, SMA is characterized by the SMN protein lack, resulting in motor neuron impairment ...
Giovanna Menduti, Daniela Maria Rasà, Serena Stanga, Marina Boido   +3 more
doaj   +1 more source

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

The Illness Narratives of Children and Young People With Spinal Muscular Atrophy: A Scoping Review

Journal of Advanced Nursing, Volume 82, Issue 9, Page 8375-8390, September 2026.
ABSTRACT Aim(s) This review seeks to explore the illness narratives of children and young people focusing on their healthcare trajectories; the right to health; and the kind of stories told about them. Design This scoping review adopts a narrative approach to analyse how the illness experience of Spinal Muscular Atrophy is represented in the literature,
Marcela González‐Agüero, Constanza Quezada, Valentina Turén, Josefa Camelio, Martina De Filippi, Caroline Bradbury‐Jones, Julie Taylor   +6 more
wiley   +1 more source

Anion‐Exchange Membrane Chromatography for the Separation of Empty and Full Adeno‐Associated Viral Capsids

Biotechnology Journal, Volume 21, Issue 8, August 2026.
Graphical Abstract and Lay Summary The anion‐exchange membrane elution chromatography for the separation of empty and full AAVs is explored and optimized. The role played by important parameters like flowrate, gradient slope, membrane loading, and feed residence time is studied in detail.
Luca Ossi, Samuele Delfino, Helena Marie, Michael Schulte, Mattia Sponchioni   +4 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  

Targeted Interventions in HAM/TSP: Emerging Therapies and Future Directions – A Narrative Review

Health Science Reports, Volume 9, Issue 8, August 2026.
ABSTRACT Background and Aim Human T‐Lymphotropic virus type 1 (HTLV‐1) infection can lead to HTLV‐1‐associated myelopathy/tropical spastic paraparesis (HAM/TSP), a debilitating chronic neurological disease characterized by progressive lower limb spasticity and autonomic dysfunction.
Meygol Mirzaei Rezaei, Mahdi Khosravi Nia, Kasra Allaei Rouzbahani, Maryam Kazemi, Negar Asghari Hosori, Mehdi Norouzi, Farzaneh Sotoudegan, Narges Eslami, Negar Ariamand, Vahid Shahnavaz, Arash Letafati, Sayed‑Hamidreza Mozhgani   +11 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

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

Journal of Market Access & Health Policy, 2021
Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results.
Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro, Omar Dabbous   +11 more
doaj   +1 more source

Exploring Genetic Therapies Targeting Amyotrophic Lateral Sclerosis in Animal Models: A Systematic Review and Meta‐Analysis

The Journal of Gene Medicine, Volume 28, Issue 8, August 2026.
Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease for which there is currently no known cure. This systematic review explores the efficacy of genetic therapies used to target ALS in preclinical studies using in vivo rodent models. Outcomes investigated include the total number of motor neurons, rodent survival, and muscle function
Hannah E. Wedgwood, Richard I. Tuxworth, Zubair Ahmed   +2 more
wiley   +1 more source

Capture of adeno‐associated viruses from clarified lysate by continuous flow ultracentrifugation: a comparative study of iodixanol and sucrose gradients

Journal of Chemical Technology &Biotechnology, Volume 101, Issue 7, Page 1384-1395, July 2026.
Abstract BACKGROUND Adeno‐Associated Virus vectors are widely used in gene therapy due to their safety and efficacy. Despite the interest in their therapeutical application, AAV purification remains a significant challenge. Density gradient ultracentrifugation using cesium chloride, sucrose, or iodixanol gradients are common methods for AAV ...
Sabrina Leigheb, Maral Rahimzadeh, Nikolaus Virgolini, Aida Tleuova, Mahbod Mousavian, Astrid Dürauer, Patricia Pereira Aguilar   +6 more
wiley   +1 more source