NIH Director’s Blog: One little girl’s story highlights the promise of precision medicine

October 24, 2019

NIH Director’s Blog reports on Mila Makovec, an 8-year-old Boston Children’s patient with Batten disease, a rapidly progressing neurological disorder that is fatal. Boston Children’s Timothy Yu, MD, PhD, and his team found her highly unusual mutation, designed an oligonucleotide drug to bypass it, engaged industry partners and launched a treatment trial — just for her. Read more at NIH Director’s Blog.