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Meta-analyses of intervention research confirm significant improvements in expressive and receptive language, social communication, and AAC use following targeted speech therapy

Preclinical data is based on animal models

The ubiquitinated HIF-1 was examined by western blot

At 2 years after starting treatment with the investigational drug nirogacestat, three-quarters of trial participants were alive without their disease getting worse, compared with less than half of patients who were given a placebo