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Nucleic Acid TherapeuticsVol. 32, No. 2 ReviewsOpen AccessCreative Commons license Abstract The drug development process is a long and arduous one, especially for rare diseases. Patient and patient representatives can and should be involved in this process from an early stage, since they have the perspective of living with a disease on a daily basis and can best identify which symptoms are the largest burden and which benefits would be more important to them.
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