Understanding what happens with new treatments

Today, we have a wonderful advantage: information about new researchs is widely available on the Internet. This information is often a source of great hope for the DMD community. But, sometimes, it’s hard to understand exactly what’s going on. Beyond what you can do for yourself or your child, what can you do as a community to be heard and to bring us closer to finding new therapies for DMD?

  • Ataluren (Translarna™) is approved within the European Union (EU) Member States, Iceland, Liechtenstein, Norway, Israel and South Korea under the trade name Translarna™ for the treatment of nmDMD in ambulatory patients aged five years and older. Translarna is the first treatment approved for the underlying cause of DMD. The European Medicines Agency, or EMA, has designated ataluren as an orphan medicinal product and the U.S. Food and Drug Administration, or FDA, has granted orphan drug designation to ataluren for the treatment of nmDMD.
  • Exondys 51 (eteplirsen) received accelerated approval in September 2016 by the U.S. Food and Drug Administration (FDA), but additional clinical testing is still required before the FDA issues a full approval. This means DMD patients amenable to exon 51 skipping may have access to the treatment in the U.S., but it is unavailable in many other countries.
  • For now, the MAP (managed access program) will be accessible to patients who live in Argentina, Brazil, Canada, Colombia, France, Germany, Greece, Iceland, Italy, Mexico, Spain, Turkey, and the United Kingdom. Link here

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