A randomized, controlled trial of ZYN002 cannabidiol transdermal gel in children and adolescents with fragile X syndrome (CONNECT-FX) - Journal of Neurodevelopmental Disorders

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A study published in the Journal of Neurodevelopmental Disorders finds that patients with Fragile X Syndrome and ≥ 90% methylation of the FMR1 gene, show a significant reduction in behavioral symptoms when treated with Zygel, a cannabidiol gel.

The primary efficacy end point was the change from baseline to week 12 in the ABC-CFXSSocial Unresponsiveness/Lethargy subscale score, and Clinical Global Impression, Improvement at week 12. Exploratory end points to support meaningful change analyses included domain specific and overall behavior Caregiver Global Impression of Severity and Change items .

]. Caregivers rate how problematic a particular behavior has been over the past week on a 4-point rating scale ranging from “not at all a problem” to “the problem is severe in degree.” The ABC-CThe CaGI-S asks caregivers to rate the overall severity of their child’s problems with social avoidance and isolation , social interactions , and irritable/disruptive behavior as well as overall behavior over the past week on a 4-point rating scale ranging from “no problems” to “severe problems.

Source: Healthcare Press (healthcarepress.net)

 

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