Join the club for FREE to access the whole archive and other member benefits.

Drug invented for a girl with Batten disease


Key points from article :

Mila Makovec, now aged eight, was diagnosed with fatal and untreatable Batten disease.

Doctors from Boston Children's Hospital in the US created the tailor-made drug for Mila.

Mila was treated with the drug - named Milasen - on 31 January 2018.

Drug sticks to the forming mRNA and stops it being cut in the wrong the place.

Details have been published in the New England Journal of Medicine.

Doctors from Boston sequenced in detail whole genome of Mila and found the disease-causing mutation

Mentioned in this article:

Health Organisation

Center for pediatric health care.


Scientific Journal devoted to medical research.


Doctor and Principal investigator of the Yu lab.