Rett  syndrome  is  a  rare  genetic  disorder  that  affects  the  way  the  brain  develops.  The  medication  trofinetide  (DAYBUE™)  was  studied  in  a  large  clinical  trial  called  LAVENDER,  where  it  showed  a  benefit  in  reducing  symptoms  of  Rett  syndrome  versus placebo (placebo did not contain medication but looked the  same  as  trofinetide  and  was  taken  in  the  same  way).  The  most  common  side  effect  in  the  trial  was  diarrhea  (frequent  and/or watery bowel movements). In order to help caregivers and  healthcare  providers,  experts  created  recommendations  on  how  to  prevent  and  manage  diarrhea  if  it  occurs  during  trofinetide treatmentThis Plain Language Summary of Publication article from Future Rare Diseases looks at using trofinetide, a medication also known as DAYBUE, in reducing symptoms of Rett syndrome. Rett syndrome is a rare genetic disorder that affects the way the brain develops. Experts created recommendations on how to prevent and manage diarrhea ( the most common side effect in the study) if it occurs during trofinetide treatment.

Read the full article here.

The original on which this summary is based is called ‘Recommendations for the management of diarrhea with trofinetide use in Rett syndrome’ and was published in Expert Opinion on Orphan Drugs. 

You can read the original article here.