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Enzyme replacement therapy
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Current data, viewed at
Yearly
resolution.
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Application
keyword
Enzyme replacement therapy
RIS
0.001
(Research Intensity Score)
Papers
91
Top papers
Aberrant upregulation of the glycolytic enzyme PFKFB3 in CLN7 neuronal ceroid lipofuscinosis
2022 · 43 citations
Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes
2020 · 71 citations
Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice
2023 · 20 citations
Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa
2025 · 12 citations
<p>Diagnosis and Screening of Patients with Fabry Disease</p>
2020 · 68 citations
Newest papers
Pompe Disease: Pathogenesis, Molecular Mechanisms, Neurological Aspects, Diagnostics and Modern Therapeutic Approaches
2026 · 2 citations
Autophagy–Lysosomal Dysfunction as a Converging Mechanism of Cardiomyopathy in Lysosomal Storage Disorders: From Pathobiology to Targeted Therapy
2026 · 1 citations
Cardiomyopathy in glycogen storage diseases: diagnosis, prognosis, and advanced management
2026 · 1 citations
Metabolomic Insights into Lysosomal Storage Diseases: An Untargeted View
2026 · 1 citations
Fabry cardiomyopathy presenting as hypertrophic phenotype with left ventricular outflow tract obstruction: a case series
2026
A sample from the 91 top papers behind Enzyme replacement therapy.
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Enzyme replacement therapy's papers
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