Associated Papers
Effect of Readthrough Treatment in Fibroblasts of Patients Affected by Lysosomal Diseases Caused by Premature Termination Codons.
Matalonga L et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics, 2015
Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases.
Gómez-Grau M et al.
Plos One, 2015
Identification and characterization of SMPD1 mutations causing Niemann-Pick types A and B in Spanish patients.
Rodríguez-Pascau L et al.
Human Mutation, 2009
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