Find a Cure
The last few years has brought unprecedented attention to Rett Syndrome by academic scientists, researchers and executives of pharmaceutical and biotech companies and more recently, life sciences investors. The interest stems from the advantages enjoyed by the Rett field: a known and single genetic cause, expectation of reversibility, NIH funded natural history study and Rett clinics with significant patient populations.
Since Rett Syndrome is caused by defective MECP2 it stands to reason that approaches that attack the root cause, MECP2 itself, hold the greatest promise of significantly improving symptoms. Therefore approaches such as activating the silent MECP2, gene therapy and protein replacement can be classified as potentially curative approaches.