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  • 1-9 ل  9 نتائج ل ""Zolgensma""

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Publisher : elsevier
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Academic Journal

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational studyResearch in context

Subjects: Spinal muscular atrophy; Gene addition therapy; SMA

  • Source: The Lancet Regional Health. Europe, Vol 47, Iss , Pp 101092- (2024)

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Academic Journal

2024 update: European consensus statement on gene therapy for spinal muscular atrophy

Subjects: Adeno-associated viral vector; Disease modifying treatment; Effectiveness

  • Source: ISSN: 1090-3798 ; European Journal of Paediatric Neurology ; https://hal.science/hal-04727214 ; European Journal of Paediatric Neurology, 2024, 51, pp.73-78. ⟨10.1016/j.ejpn.2024.06.001⟩.

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Academic Journal

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

Subjects: Gene therapy; Adeno-associated virus gene therapy; Immune responses to AAV gene therapy

  • Source: Neurotherapeutics, Vol 21, Iss 4, Pp e00435- (2024)

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Conference

SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES: P.187 Systematic literature review of the economic burden and economic evaluations in spinal muscular atrophy

Subjects: Spinal muscular atrophy; cost; economic evaluation

  • Source: Neuromuscular Disorders (2020-10); World Muscle Society, 27/09/2020 au 02/10/2020

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Academic Journal

AAV-aMTD-Parkin, a therapeutic gene delivery cargo, enhances motor and cognitive functions in Parkinson's and Alzheimer’s diseases

Subjects: Adeno-associated virus; Therapeutic-molecule systemic delivery technolog; Advanced macromolecule transduction Domain

  • Source: Pharmacological Research, Vol 208, Iss , Pp 107326- (2024)

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Academic Journal

2024 update: European consensus statement on gene therapy for spinal muscular atrophy

Subjects: Teràpia genètica; Atròfia muscular espinal - Tractament; Decisió de grup

  • Source: Scientia

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Academic Journal

Adeno-associated virus vector hydrogel formulations for brain cancer gene therapy applications

Subjects: RAAV; Gelatin matrices; Hydrogel

  • Source: Biomedicine & Pharmacotherapy, Vol 170, Iss , Pp 116061- (2024)

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Academic Journal

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors

Subjects: AAV; intrathecal; intravenous immunoglobulin

  • Source: Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 272-280 (2022)

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Academic Journal

SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES: P.191 Preliminary data for the cost-effectiveness assessment of the newborn screening for SMA in Belgium

Subjects: Newborn screening; Spinal muscular atrophy; Human health sciences

  • Source: Neuromuscular Disorders (2020-10)

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  • 1-9 ل  9 نتائج ل ""Zolgensma""