Exondys 51: On September 19th 2017, Exondys 51 was granted Accelerated Approval by the Food and Drug Administration (FDA). It is the only FDA approved therapy for the treatment of Duchenne. Exondys 51 is not approved by the European Medicines Agency (EMA). Exondys 51 treats approximately 13% of the Duchenne population who are amenable to the skipping of exon 51 on the dystrophin gene. If you or someone you know may be amenable to Exondys 51, you should contact Sarepta Therapeutics, the manufacturer of Exondys 51, for more information on how to get started.
Translarna: On May 23rd, 2014, Translarna was granted Conditional Approval by the EMA. It is the only EMA approved therapy for the treatment of Duchenne. Translarna is has not been approved by the FDA for commercial use in the United States. Translarna treats the approximately 13% of the Duchenne population who have a premature stop codon mutation, also called a nonsense mutation, on the dystrophin gene. If you or someone you know may be amenable to Translarna, you should talk with your doctor.