Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.

Rosenberg, Jonathan B; Chen, Alvin; Kaminsky, Stephen M; Crystal, Ronald G; Sondhi, Dolan

Rosenberg, Jonathan B; Chen, Alvin; Kaminsky, Stephen M; Crystal, Ronald G; Sondhi, Dolan.

Afiliación

Rosenberg JB; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.
Chen A; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.
Kaminsky SM; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.
Crystal RG; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.
Sondhi D; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.

Expert Opin Orphan Drugs ; 7(11): 473-500, 2019.

Article en En | MEDLINE | ID: mdl-33365208

Palabras clave

Neuronal ceroid lipofuscinoses (NCL); adeno-associated virus (AAV); ceroid lipofuscinosis, neuronal (CLN); clinical trials; enzyme replacement therapy (ERT); gene therapy; palmitoyl protein thioesterase-1 (PPT1); tripeptidyl peptidase-1 (TPP1)

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Expert Opin Orphan Drugs Año: 2019 Tipo del documento: Article Pais de publicación: Reino Unido

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