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Authors
Amedro, Pascal https://orcid.org/0000-0003-3649-0294
Andrianoely, Mathieu https://orcid.org/0009-0007-1025-7909
Gohier, Pauline https://orcid.org/0009-0003-4258-9204
Leroux, Justine
Aladjidi, Nathalie https://orcid.org/0000-0003-0231-4460
Lagorce, Stephanie
Daccord, Melissa
Audie, Marion
Botte, Astrid
Nubret, Karine https://orcid.org/0009-0006-7556-8081
Richard, Pascale https://orcid.org/0000-0002-2390-3005
Roche, Sandrine
Lamireau, Delphine
Tetard, Candice
Trimouille, Aurelien https://orcid.org/0000-0002-3457-5684
Desir, Arielle https://orcid.org/0009-0008-4073-323X
Mesli, Samir
Donadieu, Jean https://orcid.org/0000-0002-4485-146X
Gotchac, Julien https://orcid.org/0000-0001-7744-7028
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Article History
Received: 3 October 2025
Accepted: 26 June 2026
First Online: 7 July 2026
Declarations
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: The study was conducted in accordance with the Declaration of Helsinki. Compassionate use of elamipretide was approved by the French Drug Administration (ANSM) within the Rare Disease Program. Written informed consent for treatment and participation in the study was obtained from the patient’s parents.
: Written informed consent for publication of this case report and the accompanying clinical images was obtained from the patient’s parents.
: The authors declare that they have no competing interests. The manufacturer of elamipretide was not involved in the preparation, review, or approval of this manuscript.
