Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec –A Single Centre Experience

BACKGROUND: Recently gene therapy with onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy (SMA). As the experience from clinical trials is limited, there are still uncertainties for which patient population the treatment can be considered safe and effective. METH...

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Detalles Bibliográficos
Autores principales: Friese, Johannes, Geitmann, Stephanie, Holzwarth, Dorothea, Müller, Nicole, Sassen, Robert, Baur, Ute, Adler, Kristin, Kirschner, Janbernd
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8075402/
https://www.ncbi.nlm.nih.gov/pubmed/33427694
http://dx.doi.org/10.3233/JND-200593

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8075402/
https://www.ncbi.nlm.nih.gov/pubmed/33427694
http://dx.doi.org/10.3233/JND-200593

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