Evaluation of Systemic Gentamicin as Translational Readthrough Therapy for a Patient With Epidermolysis Bullosa Simplex With Muscular Dystrophy Owing to PLEC1 Pathogenic Nonsense Variants.
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M. del Río | F. Larcher | Marta García | D. Woodley | R. D. de Lucas | M. Escámez | S. Guerrero-Aspizua | M. C. D. de Arriba | L. Quintana | B. Duarte | J. A. Membrilla | L. Martínez-Santamaría | J. Mascías | R. Maseda | C. Hernández-Fernández | N. Illera | I. Esteban‐Rodríguez | Alberto Iglesias Sigüenza | J. Membrilla | Mariá C de Arriba