Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome.
Heather M O'LearyWalter E KaufmannKatherine V BarnesKshitiz RakeshKush KapurDaniel C TarquinioNicole G CantwellKatherine J RocheSuzanne A RoseAlexandra C WalcoNatalie M BruckGrace A BazinIngrid A HolmMark E AlexanderLindsay C SwansonLauren M BaczewskiChi‐Sang PoonJuan M Mayor TorresCharles A NelsonMustafa SahinPublished in: Annals of clinical and translational neurology (2018)
As in the phase 1 trial, rhIGF-1 was safe; however, the drug did not reveal significant improvement, and some parameters worsened.