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UNC Health Advances Gene Therapy Research for Rett Syndrome
UNC Health reports progress in its gene therapy research aimed at treating Rett syndrome, a rare neurological disorder that primarily affects girls and causes severe impairments in movement, communication and daily function. The announcement highlights the health system's ongoing work to develop genetic treatments for conditions that currently have limited therapeutic options, drawing attention from families and researchers following the rare disease field.
Why now: Families and researchers affected by Rett syndrome closely follow any progress toward a potential gene therapy treatment.
Evidence
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