The Chemical Compound PTC124 Does Not Affect Cellular Electrophysiology of Cardiac Ventricular Myocytes

Repairing faulty genes by aminoglycosides: Development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations

(2010) Igor Nudelman et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Readthrough of nonsense mutations in Rett syndrome: evaluation of novel aminoglycosides and generation of a new mouse model

(2010) Cornelia Brendel et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Drug-Induced Long QT Syndrome

(2010) P. Kannankeril et al.   PHARMACOLOGICAL REVIEWS

Readthrough of nonsense mutation W822X in the SCN5A gene can effectively restore expression of cardiac Na+ channels

(2009) Siyong Teng et al.   CARDIOVASCULAR RESEARCH

An international compendium of mutations in the SCN5A-encoded cardiac sodium channel in patients referred for Brugada syndrome genetic testing

(2009) Jamie D. Kapplinger et al.   HEART RHYTHM

Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

(2009) Igor Nudelman et al.   JOURNAL OF MEDICINAL CHEMISTRY

Psychosocial Considerations for Children and Young Adolescents with Implantable Cardioverter Defibrillators: An Update

(2009) SAMUEL F. SEARS et al.   PACE-PACING AND CLINICAL ELECTROPHYSIOLOGY

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

(2008) Eitan Kerem et al.   LANCET

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

(2008) M. Du et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA