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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 [PDF]

The Lancet Regional Health. Europe
Summary: Background: Real-world data on gene addition therapy (GAT) with onasemnogene abeparvovec (OA), including all age groups and with or without symptoms of the disease before treatment are needed to provide families with evidence-based advice and ...
Claudia Weiß +308 more
doaj +2 more sources

Issue Information

International Wound Journal, Volume 23, Issue 6, June 2026.
wiley +11 more sources

Cancer in rural America: Improving access to clinical trials and quality of oncologic care. [PDF]

CA Cancer J Clin
Abstract Individuals from rural areas in the United States suffer higher rates of morbidity and mortality from cancer than their urban counterparts. This review is based on the idea that equity—the elimination of unnecessary and preventable differences between groups of individuals—should underlie access to cancer care resources for patients from rural
Unger JM, McAneny BL, Osarogiagbon RU.
europepmc +2 more sources

Revisão sistemática da eficácia e segurança do onasemnogeno abeparvoveque para tratamento de pacientes com Atrofia Muscular Espinhal tipo 1

Jornal de Assistência Farmacêutica e Farmacoeconomia, 2023
Introdução: A atrofia muscular espinhal 5q (AME) é uma doença genética rara, caracterizada por uma progressiva perda de força muscular decorrente da degeneração de neurônios da medula espinhal. A AME tipo I corresponde a aproximadamente 58% dos casos de
Stéfani Sousa Borges +6 more
doaj +1 more source

Elective removal vs. retaining of hardware after osteosynthesis in asymptomatic patients—a scoping review

Systematic Reviews, 2020
Background Osteosynthesis is the internal fixation of fractures or osteotomy by mechanical devices (also called hardware). After bone healing, there are two options: one is to remove the hardware, the other is to leave it in place.
Barbara Prediger +3 more
doaj +1 more source

Remote Psychotherapy during the COVID-19 Pandemic: A Mixed-Methods Study on the Changes Experienced by Austrian Psychotherapists

Life, 2023
The outbreak of the COVID-19 pandemic and associated measures to contain the SARS-CoV-2 coronavirus required a change in treatment format from face-to-face to remote psychotherapy.
Michael Stadler +10 more
doaj +1 more source

Identification of novel proteins and mRNAs differentially bound to the Leishmania Poly(A) Binding Proteins reveals a direct association between PABP1, the RNA-binding protein RBP23 and mRNAs encoding ribosomal proteins.

PLoS Neglected Tropical Diseases, 2021
Poly(A) Binding Proteins (PABPs) are major eukaryotic RNA-binding proteins (RBPs) with multiple roles associated with mRNA stability and translation and characterized mainly from multicellular organisms and yeasts.
Ludmila A Assis +11 more
doaj +2 more sources

Comunicação breve: Revisão Sistemática da eficácia e segurança do onasemnogeno abeparvoveque para tratamento de pacientes com Atrofia Muscular Espinhal Tipo 1

Jornal de Assistência Farmacêutica e Farmacoeconomia, 2023
Objetivo: Foram avaliadas evidências de eficácia e segurança do onasemnogeno abeparvoveque no tratamento da atrofia muscular espinhal tipo 1. Método: Foi conduzida uma revisão sistemática da literatura, conforme protocolo aprovado previamente ...
Stéfani Sousa Borges +6 more
doaj +1 more source

Factors predicting failure of internal fixations of fractures of the lower limbs: a prospective cohort study

BMC Musculoskeletal Disorders, 2021
Background We assessed predictive factors of patients with fractures of the lower extremities caused by trauma. We examined which factors are associated with an increased risk of failure.
Barbara Prediger +7 more
doaj +1 more source

Estudo de custo-efetividade para o tratamento da AME tipo 1

Jornal de Assistência Farmacêutica e Farmacoeconomia, 2023
Introdução: O desenvolvimento de tecnologias inovadoras para o tratamento da Atrofia Muscular Espinhal (AME) tipo 1 tem se mostrado promissor na mudança da morbimortalidade dos pacientes com essa condição.
Fernanda d Athayde Rodrigues +6 more
doaj +1 more source

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