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Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

103

Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

100

A Multistakeholder Perspective on Advancing Individualized Therapeutics

90

Illustrated Examples

The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study

57

A framework for individualized splice-switching oligonucleotide therapy

214

OTS Rare Disease N-of-1+ Briefing Document, Jan 2020

57

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