Timothy Yu, Boston Children's Hospital attending physician, division of genetics and genomics

How many pa­tients could ben­e­fit from cus­tom ge­net­ic ther­a­pies? Tim­o­thy Yu nar­rows in on an an­swer

Tim­o­thy Yu sent shock­waves through the ge­net­ic ther­a­py world four years ago af­ter re­veal­ing that he had de­vel­oped — in un­der a year — a treat­ment tai­lored to a young girl’s unique ge­net­ic mu­ta­tion.

That drug, dubbed Mi­lasen in hon­or of the girl Mi­la, raised the bar on how tru­ly per­son­al­ized a med­i­cine could be, and ig­nit­ed ef­forts to de­vel­op the so-called n-of-1 ther­a­pies for more pa­tients. Yet there was one ques­tion that kept nag­ging at Yu: How many oth­ers like Mi­la were out there?

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.