カテゴリー: アンケート

調査結果：世界のMECP2重複研究コミュニティが語る

Research on MECP2 Duplication Syndrome has taken an unprecedented step: entering the world of clinical trials. This is a spark of hope for many families who have worked tirelessly for years to provide a better future for their children. With the launch of early-stage clinical trials such as ATTUNE (ION440 by Ionis) in the U.S. and HERO (HG204 by HuidaGene) in China, we are witnessing a historic moment in the search for a treatment. These phase 1/2 trials will help determine whether these investigational drugs are safe and potentially effective – but they are only the beginning. To prepare for what comes next – including larger, later-phase trials – we […]