mILASEN - hOW IT WORKS

 
Mila Makovec's CLN7 Batten disease is caused by a unique and unusual genetic mutation. Learn how the mutation damages Mila's nerve cells and how an oligonucleotide drug, designed at Boston Children's Hospital specially for her, fixes her gene.
 

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. Mila’s fight was not in time for her, but her story is giving enormous hope to many more across all 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 individualized medicines. We believe that by making this platform approach accessible across all rare diseases, we can help solve the drastic problem of rare disease in children. Our hope is that our work will give children like Mila a second chance at life.

a new path of truly personal medicines

 

the path to a brighter future for many

Mila was once like any happy, healthy child. But at age 3, she started to falter. At 6, she was diagnosed with Batten disease, a rare and fatal genetic disorder. She lost her sight, her language and her ability to walk independently.
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WE NEED YOUR HELP NOW

Mila was given an unprecedented second chance at life through the first-ever truly personal medicine, Milasen. Now we need your help to pave this path for many more children like Mila across hundreds of rare diseases.

Donate now

Mila's Miracle Foundation, Inc. is a 501(c)(3) nonprofit organization. All donations are tax-deductible.