Neuren Media and Analyst Coverage, page-1261

Here’s something to brighten up everyone’s day after a less than stellar week. It has nothing to do with media or analyst coverage. It’s better.

A newly published paper by three US-based paediatric neurology specialists presents three case studies of Rett syndrome patients who have been treated with trofinetide.

Improvement in symptoms of Rett syndrome with trofinetide beyond clinical trial efficacy assessments: case reports

In clinical trials, trofinetide improved the symptoms of RTT, yet some symptoms that are relevant to the syndrome were not studied. Here, we present the anecdotal, real-world experience of three female patients with RTT who experienced improvement in symptoms of the syndrome not covered in clinical trials: improvement in verbal skills, reduction in the frequency of seizures and improvement in motor skills.

https://www.tandfonline.com/doi/full/10.1080/23995270.2024.2375191#summary-abstract