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signatures. Finally, there is a critical need to develop therapeutic strategies beyond pharmaceutical
interventions including early pain interventions, physical and occupational rehabilitation, psychosocial
support groups, and integrative care models to fully address the needs of individuals with NF1-associated
PNs across their life span.

Preclinical work suggests that restoring the missing neurofibromin gene could reduce PN burden and
alleviate symptoms . In an NF1 xenograft model, an engineered adeno-associated virus for NF (AAV-NF)
[24]
(K55) vector showed reduced hepatic uptake and enhanced tumor targeting, improving intratumoral
delivery bringing gene therapy a step closer to first-in-human clinical trials . Sustained collaboration among
[24]
patients, researchers, and philanthropic partners, such as the Children’s Tumor Foundation and the Gilbert
Family Foundation, will accelerate the development of these promising treatment strategies and may
ultimately lead to a cure for NF1.

DECLARATIONS
Authors’ contribution
Original draft preparation, reviewing, editing: Kim H
Assisted with draft preparation, reviewing and editing: Polen J
Assisted with draft preparation, supervision, reviewing, and editing: Kaur G

Availability of data and materials
Not applicable.

AI and AI-assisted tools statement
During the preparation of the manuscript, the authors used ChatGPT (OpenAI) solely for language clarity
and readability. After using this tool, the authors reviewed and edited the content and take full responsibility
for the content of the published article.

Financial support and sponsorship
None.

Conflicts of interest
Gurcharanjeet Kaur is a Guest Editor Assistant of the Special Issue "Topic: Advances in Neurofibromatosis -
The Future is Bright" of the journal Rare Disease and Orphan Drugs. Gurcharanjeet Kaur was not involved in
any steps of the editorial process, notably including reviewers' selection, manuscript handling, or
decision-making. The other authors declare that there are no conflicts of interest.

Ethical approval and consent to participate
Not applicable.

Consent for publication
Not applicable.

Copyright
© The Author(s) 2026.

REFERENCES
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