being the first

Mila is the first person in the world to receive a drug customized for just one person. It was named Milasen. Although Mila had lost a lot before she began treatment, she was given an unprecedented second shot at life. We still don’t know what Mila’s future holds, but we do know that her fight is giving enormous hope to so many more across hundreds of rare diseases that otherwise would have no chance.

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mila’s gift to the world

Milasen gave Mila hope that we never thought was possible. But there are still millions of children around the world dying from genetic diseases like Batten.

We are on a mission to turn Mila’s incredible story into an entirely new treatment path of truly personal medicines. We are working to foster this new field and eventually make it accessible to many more across hundreds of rare diseases. Our hope is that our work will give Mila and many others like her a second chance at life.

a new path of truly personal medicines

the path to a brighter future for many