Rare Daily Staff

Mila Makovec, a young girl who was the recipient of a custom antisense oligonucleotide therapy to treat her fatal ultra-rare disease, and excited the rare disease community with the potential for bespoke therapies, died. She was 10 years old.

Makovec had CLN7, an ultra-rare form of the neurodegenerative condition Batten disease. Julia Vitarello, her mother, and founder and CEO of Mila’s Miracle Foundation, announced her death online in a post, dated February 12.

“Yesterday, Mila’s spirit left her body. A day so trying that it will forever change me. Just as I labored to give birth to her warm body as it worked its way so naturally into the world ten years ago, yesterday Mila labored in my arms and Alek’s to release her spirit and work her way so naturally through the process of letting her body go,” Vitarello wrote. “I watched as she found peace, free of the pain and confinement, free to fly as the hummingbird Azlan has seen her move into. Today, I find myself on my knees outside Mila’s bedroom door, unable to go in, my head buried in the carpet of tears. I gasp in pain, unable to find her, to hold her. Then I sit up and breathe, and think about this next chapter in my life.”

Boston Children’s Hospital researcher Timothy Yu developed milasen, an experimental antisense oligonucleotide specifically to treat Mila in January 2018. The drug was developed and delivered in less than a year and inspired others to pursue bespoke antisense and gene therapies to treat other children with ultra-rare conditions.

 

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