English (United Kingdom)

https://curated-unify.zendy.io/wp-json/zendy-region/v1/featured_content/oa?rat=en

https://curated-unify.zendy.io/wp-json/zendy-region/v1/highlighted_journal/

Zendy Plus

Presents the access of premium content as premium feature

Premium Content

Presents the keyphrase highlighting as premium feature

Keyphrase Highlighting

Presents the summarisation as premium feature

Summarisation

Insights

Presents the pdf analysis as premium feature

PDF Analysis

Presents the zaia usage as premium feature

ZAIA

Zendy Tools

Zendy Open

Objective  To measure the efficacy of mecasermin (recombinant human insulin‐like growth factor 1, rh IGF ‐1), for treating symptoms of Rett syndrome ( RTT ) in a pediatric population using a double‐blind crossover study design.    Methods  Thirty girls with classic  RTT  in postregression stage were randomly assigned to placebo or rh IGF ‐1 in treatment period 1 and crossed over to the opposite assignment for period 2 (both 20 weeks), separated by a 28‐week washout period. The primary endpoints were as follows: Anxiety Depression and Mood Scale ( ADAMS ) Social Avoidance subscale, Rett Syndrome Behaviour Questionnaire ( RSBQ ) Fear/Anxiety subscale, Parent Target Symptom Visual Analog Scale ( PTSVAS ) top three concerns, Clinical Global Impression ( CGI ), Parent Global Impression ( PGI ), and the Kerr severity scale. Cardiorespiratory‐ and electroencephalography ( EEG )‐based biomarkers were also analyzed.    Results  There were no significant differences between randomization groups. The majority of  AE s were mild to moderate, although 12 episodes of serious  AE s occurred. The Kerr severity scale,  ADAMS  Depressed Mood subscale, Visual Analog Scale Hyperventilation, and delta average power change scores significantly increased, implying worsening of symptoms. Electroencephalography ( EEG ) parameters also deteriorated. A secondary analysis of subjects who were not involved in a placebo recall confirmed most of these findings. However, it also revealed improvements on a measure of stereotypic behavior and another of social communication.    Interpretation  As in the phase 1 trial, rh IGF ‐1 was safe; however, the drug did not reveal significant improvement, and some parameters worsened.

Placebo‐controlled crossover assessment of mecasermin for the treatment of Rett syndrome