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.
The therapy met its primary endpoints in two Phase 3 studies and offers a new approach for the tens of millions of people living with the psychiatric condition.
Scores of neurons are already dead by the time Parkinson’s disease is usually diagnosed. Scientists aim to identify patients earlier and intervene before it’s too late.