Arbaclofen in fragile X syndrome: results of phase 3 trials

@inproceedings{BerryKravis2016ArbaclofenIF,
  title={Arbaclofen in fragile X syndrome: results of phase 3 trials},
  author={Elizabeth M. Berry-Kravis and Randi Hagerman and Jeannie Visootsak and Dejan B. Budimirovic and Walter E. Kaufmann and Maryann Cherubini and Peter Zarevics and Karen L Walton-Bowen and Paul P. Wang and Mark F. Bear and Randall L. Carpenter},
  booktitle={Journal of Neurodevelopmental Disorders},
  year={2016}
}
Arbaclofen improved multiple abnormal phenotypes in animal models of fragile X syndrome (FXS) and showed promising results in a phase 2 clinical study. The objective of the study is to determine safety and efficacy of arbaclofen for social avoidance in FXS. Two phase 3 placebo-controlled trials were conducted, a flexible dose trial in subjects age 12–50 (209FX301, adolescent/adult study) and a fixed dose trial in subjects age 5–11 (209FX302, child study). The primary endpoint for both trials… CONTINUE READING
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