After tackling toxicity and a trial halt, Elena Koundourakis and her team at Takeda are on the cusp of a first-in-class drug for people living with narcolepsy.
Rett syndrome currently has no cure. Now, innovations in gene therapy bring the possibility of treating the root cause of the disease closer than ever before.
The enzyme PGK1 sets the rate of cellular energy production and may be an ideal drug target when Parkinson’s disease neurons struggle to fuel themselves.