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Addressing the Needs of Nano-Rare Patients.
Nucleic Acid Ther
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n-Lorem Foundation, Carlsbad, California, USA.
Progress in molecular biology and translational science addressing the needs of nano-rare patients.
Prog Mol Biol Transl Sci
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n-Lorem Foundation, Carlsbad, CA, United States. Electronic address:
The healthcare systems in the developed economies were established primarily to address the more prevalent diseases and have been customized to support the provision of therapeutics to rare patients. However, with the ever-broader implementation of genomic sequencing, it is clear that there are significantly more disease-causing mutations in the human genome than realized and that many mutations are much rarer than current definitions of rare disease populations. Given this, I propose parsing patient populations and defining patient populations more precisely.
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