Off-Label Use of Ataluren in Four Non-ambulatory Patients With Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength

• Main Authors: Daniel Ebrahimi-Fakhari (Author), Ulrich Dillmann (Author), Marina Flotats-Bastardas (Author), Martin Poryo (Author), Hashim Abdul-Khaliq (Author), Mohammed Ghiath Shamdeen (Author), Bernhard Mischo (Author), Michael Zemlin (Author), Sascha Meyer (Author)
• Format: Book
• Published: Frontiers Media S.A., 2018-10-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

About 15% of Duchenne muscular dystrophy (DMD) cases are caused by point mutations leading to premature stop codons and disrupted synthesis of the dystrophin protein. Stop codon read-through therapy is available with the drug Ataluren (Translarna® by PTC Therapeutics). Following positive results in...