Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Barrett AM, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: long-term safety and efficacy results of the 32-month, open-label LILAC-2 study. Med. 2024 Oct 11;5(10):1275-1281. doi: 10.1016/j.medj.2024.06.007
Raspa M, Sacco P, Candrilli SD, Bishop E, Petrillo J. Validity of a condition specific outcome measure for fragile X syndrome: the Aberrant Behaviour Checklist-utility index. J Intellect Disabil Res. 2016 Sep;60(9):844-55. doi: 10.1111/jir.12264