Industry-Sponsored N-of-1 Trials in Orphan Drug Development
On Biostatistics and Clinical Trials,
N-of-1 clinical trial is often mentioned as one of the approaches to address the challenging issues in orphan drug development.
RNA therapeutic news roundup Q1 2021
CureFFI.org,
Just like back in January, I find that I’ve been accumulating a short list of interesting news updates from RNA therapeutics…
Anastasia Khvorova: oligonucleotide therapeutics, the future of human medicine
CureFFI.org,
These are my notes from Dr. Anastasia Khvorova’s lecture at the Broad Institute’s New Therapeutic Modalities Workshop series on…
One Little Girl’s Story Highlights the Promise of Precision Medicine
NIH Director's Blog,
Caption: Mila with researcher Timothy Yu and her mother Julia Vitarello. Mila’s head is covered in gauze because she’s…