Novel Drug Promising for Life-Threatening Rett Syndrome in Kids was featured yesterday as “Recommended Reading” on the Medscape website.
Medscape is a leading online global website for physicians and healthcare professionals that provides the latest medical news and expert perspectives as well as clinical information and resources.
The three page article follows the recent publication in Neurology of Phase 2 paediatric study results for trofinetide in Rett syndrome.
One independent expert quoted in the article commented that the study was well designed and the results were promising as trofinetide appeared to have a strong effect on the outcome measures that were assessed. He also commended the researchers for taking the time before the clinical trial to develop outcome measures that suited their population… “these provided a window into the disease that would not have been available with things like MRI or other outcome measures."
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