Document is current

Article History

Received: 14 January 2026

Accepted: 12 March 2026

First Online: 2 April 2026

Declarations

:

: This study involved human participants in a compassionate use program, which in Spain are individually authorized by the Spanish Agency for Medicines and Medical Devices (AEMPS) and by the ethics committee of the hospital, upon obtaining written informed consent from each patient. Compassionate use authorizations are granted by the AEMPS when a clinical trial of the same investigational product is ongoing, as was the case here. The referenced clinical trial (EudraCT 2008-000364-16; NCT01844661) was approved by the Institutional Review Board of Hospital Universitario Niño Jesús and conducted in accordance with the Declaration of Helsinki and Good Clinical Practice guidelines. All animal protocols were approved by the pertinent ethical committees and carried out in accordance with the guidelines of the European directives and Spanish laws.

: All authors have read and approved the final manuscript and consent to its publication.

: The authors declare no competing interests.