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The more people we reach, the more our impact can grow.

In 1997, we were founded to make a big difference in small patient populations. Now we seek to make an even greater impact by applying the same science-driven, patient-forward approach that propelled our last 25 years to larger genetic disorders, as well as genetic subsets of more common conditions. The end goal has always been better lives and now we can reach more.

Show Your Rare. Show You Care.
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Our Products

We find tomorrows.

Each of our approved therapies was driven by the goal of resetting the standard of care for the conditions they treat.

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