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A 6-month music therapy program improved social interaction and communication skills and reduced seizure occurrence among Rett syndrome patients, as well as relieving parental stress, a new study showed. The research article, “The effectiveness of music therapy for individuals with Rett syndrome and their families,” was published…

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…

After the U.S. Food and Drug Administration recently approved an investigational new drug application for Anavex 2-73, Anavex Life Sciences is proceeding with its Phase 2 clinical trial of the medication in Rett syndrome patients, the company announced. The trial (NCT03758924) will assess the safety, tolerability,…