A call to arms against ultra-rare diseases

Nature Biotechnology
Volume 39 Issue 6, June 2021
https://www.nature.com/nbt/volumes/39/issues/6

 

Comment | 04 June 2021
A call to arms against ultra-rare diseases
The not-for profit foundation n-Lorem seeks to provide experimental antisense oligonucleotide treatments to patients with ultra-rare disease free for their entire lifetime.
Stanley T. Crooke