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Every patient has a story.

The "I'm Not Average" campaign features fellow Americans whose lives have been impacted by treatments and cures for debilitating, chronic, or rare diseases.

< View All Stories


I'm Not Average: Ellie

Condition:
Leukodystrophy-LBSL


Age:
7


Location:
Arlington, VA


That little Ellie’s feet hurt when she ran revealed a parent’s nightmare: she had a very rare mitochondrial disease known as Leukodystrophy-LBSL. It has no current cure. #ImNotAverage


But there is hope in the form of two phase II clinical trials underway that could help Ellie and the tens of thousands of other children and adults fighting mitochondrial disease.

That hope – and innovative medical care – has Ellie back on her feet, running toward a future that could be very bright, indeed.

Condition:
Leukodystrophy-LBSL


Age:
7


Location:
Arlington, VA

connect with PhRMA...

Make your voice heard. Join the important discussion on the value and economic impact of new medicines.


connect with PhRMA...

Make your voice heard. Join the important discussion on the value and economic impact of new medicines.


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