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Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives

Antisense oligonucleotide (ASO) therapies present a promising disease-modifying treatment approach for rare neurological diseases (RNDs). However, the current focus is on “more common” RNDs, leaving a large share of RND patients still without prospect of disease-modifying treatments. In response to...

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Detalles Bibliográficos
Autores principales:	Synofzik, Matthis, van Roon-Mom, Willeke M.C, Marckmann, Georg, van Duyvenvoorde, Hermine A., Graessner, Holm, Schüle, Rebecca, Aartsma-Rus, Annemieke
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2022
Materias:	Issues in Development
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9058873/ https://www.ncbi.nlm.nih.gov/pubmed/34591693 http://dx.doi.org/10.1089/nat.2021.0039

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