Fragile X research is at a turning point. Two late-stage clinical trials, zatolmilast (EXPERIENCE) and ZYN002 (RECONNECT), could soon deliver the first approved treatments. FRAXA is also funding innovative approaches like ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.
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Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids
Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA.
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