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A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

BACKGROUND: Sanfilippo type B is a mucopolysaccharidosis (MPS) with a major neuronopathic component characterized by heparan sulfate (HS) accumulation due to mutations in the NAGLU gene encoding alfa-N-acetyl-glucosaminidase. Enzyme replacement therapy for neuronopathic MPS requires efficient enzyme...

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Detalles Bibliográficos
Autores principales:	Muschol, Nicole, Koehn, Anja, von Cossel, Katharina, Okur, Ilyas, Ezgu, Fatih, Harmatz, Paul, de Castro Lopez, Maria J., Couce, Maria Luz, Lin, Shuan-Pei, Batzios, Spyros, Cleary, Maureen, Solano, Martha, Nestrasil, Igor, Kaufman, Brian, Shaywitz, Adam J., Maricich, Stephen M., Kuca, Bernice, Kovalchin, Joseph, Zanelli, Eric
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2023
Materias:	Clinical Medicine
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9843052/ https://www.ncbi.nlm.nih.gov/pubmed/36413418 http://dx.doi.org/10.1172/JCI165076

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