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Page 44 Braun. J Transl Genet Genom. 2025;9:35-47 https://dx.doi.org/10.20517/jtgg.2024.79

Figure 1. Current pipeline of gene-based therapies. Positioning of the antisense (beige) or gene-replacement (green) therapies,
according to the stage of development (left column). Bold: drug name including commercial name (®); US: approved in USA; J: approved
in Japan; Logos: name of the sponsor; Number in parentheses: targeted exon; *: clinical hold by FDA; µdys: microdystrophin; Fab-PMO:
antibody-conjugated Phosphorodiamidate Morpholino Oligomers. Two other studies carried out in China (NCT06114056 relative to an
AAV-microdystrophin, and NCT06594094 relative to a CRISPR-gene editing approach) are not indicated here. PMO:
Phosphorodiamidate morpholino oligomer; AAV: Adeno-associated virus; FDA: Food and drug administration.

remains: the development of an economically viable model that guarantees access to these expensive
combination biological treatments for all patients , including non-ambulant individuals with DMD, BMD,
[73]
rare female DMD patients, and affected female carriers. Part of the answer relies on scientific and
technological progress.

DECLARATIONS
Acknowledgments
The author thanks the Genethon team, AFM-Telethon, and the many external collaborators, including
researchers, clinicians, and non-clinicians.

Authors’ contributions
The author contributed solely to the article.

Availability of data and materials
Not applicable.

Conflicts of interest
Genethon is a non-profit institute established by AFM-Telethon, a patient organization.

Financial support and sponsorship
Not applicable.

Ethical approval and consent to participate
Not applicable.