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Scherman. Rare Dis Orphan Drugs J 2023;2:12 https://dx.doi.org/10.20517/rdodj.2023.01 Page 29 of 35

the patient served as the basis for launching the N-of-1 clinical trial of Milasen within 1 year after first
contact with the patient.

More N-of-1 ASOs drugs have been developed through accelerated regulatory pathways for specific forms
[3]
of ataxia-telangiectasia and amyotrophic lateral sclerosis (ALS) . It is worth mentioning that gene editing is
also progressing to treat n-of-1 diseases, such as in the case of a rare mutation of Duchenne muscular
dystrophy . Treating N-of-1 diseases by personalized therapy, which was unthinkable a few years ago, is
[182]
now becoming a reality with the availability of well mastered technological platforms which drastically
reduce development duration and costs. This personalized medicine perspective for N-of-1 patients might
represent the hallmark of the ongoing genetic drug revolution. However, this will necessitate solving the
main obstacle to the generalization of RNA drug use, which consists in identifying efficient delivery
methods to all tissues.

DECLARATIONS
Authors’ contributions
The author contributed solely to the article.

Availability of data and materials
Not applicable.

Financial support and sponsorship
This work was supported by AMMF - The Cholangiocarcinoma Charity and by Sapienza University of
Rome-Fondi di Ateneo.

Conflicts of interest
All authors declared that there are no conflicts of interest.

Ethical approval and consent to participate
Not applicable.

Consent for publication
Not applicable.

Copyright
© The Author(s) 2023.

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