Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations
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R. Schüle | M. Synofzik | W. V. van Roon-Mom | H. Graessner | C. Siezen | A. Aartsma-Rus | Britt Duijndam | M. Lauffer | Tineke Coenen-de Roo