WERE YOUR PATIENTS’ GENETIC TESTS PERFORMED USING A DIFFERENT METHOD? A NEW TEST IS REQUIRED TO ACCURATELY DETERMINE EXON 51 AMENABILITY.2
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EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.
EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics Inc; October 2018.
Aartsma-Rus A, Ginjaar IB, Bushby K. The importance of genetic testing for Duchenne muscular dystrophy. J Med Genet. 2016;0:1-7.
den Dunnen JT. Leiden Muscular Dystrophy pages. Leiden, Netherlands: Center for Human and Clinical Genetics, Leiden University Medical Center; 2003. Available at dmd.nl. Accessed June 1, 2018.
Fletcher S, Adams AM, Johnsen RD, et al. Mol. Ther. 2010;18(6): 1218-1223.
Data on file.
Aartsma-Rus A, et al. Theoretic applicability of anitsense-mediated exon skipping for Duchenne muscular dystrophy mutations. Human Mutation. 2009;X:1-7.