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Objectif : Examiner la sécurité, la tolérabilité, la pharmacocinétique et l'efficacité clinique préliminaire du nusinersen intrathécal (anciennement ISIS-SMN Rx), un oligonucleotide antisens conçu pour modifier l'épissage de l'ARNm SMN2, chez des patients atteints d'atrophie musculaire spinale (SMA) infantile.
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Claudia A. Chiriboga
Università Cattolica del Sacro Cuore
Kathryn J. Swoboda
Massachusetts General Hospital
Basil T. Darras
Università Cattolica del Sacro Cuore
Neurology
The University of Texas Southwestern Medical Center
University of Utah
Boston Children's Hospital
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Chiriboga et al. (Jeudi) ont étudié cette question.
synapsesocial.com/papers/69d7370bf07a12db70b8a5af — DOI: https://doi.org/10.1212/wnl.0000000000002445
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