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Charting a path to personalized treatment for patients with orphan diseases: diagnosis of a rare, fatal neurodegenerative condition (Battens disease) led to design of a splice-modulating antisense oligonucleotide drug tailored for one patient #NBTHighligh

09:17 EDT 11 Oct 2019 | Nature Publishing

Charting a path to personalized treatment for patients with orphan diseases: diagnosis of a rare, fatal neurodegenerative condition (Battens disease) led to design of a splice-modulating antisense oligonucleotide drug tailored for one patient http://bit.ly/2M8ER5S 

Original Article: Charting a path to personalized treatment for patients with orphan diseases: diagnosis of a rare, fatal neurodegenerative condition (Battens disease) led to design of a splice-modulating antisense oligonucleotide drug tailored for one patient #NBTHighligh

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