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PTC124 treatment over 3 months improves pharmacodynamic and clinical parameters in patients with nonsense-mutation-mediated CF
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2008
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Gene TherapiesPtc124 TreatmentNonsense-mutation-mediated CfClinical TranslationGenetic DisorderMedicineImmunologyPharmacologyPathologyMolecular DiagnosticsClinical ParametersNovel TherapyVariant Interpretation
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