A Phase 3 Clinical Trial in Children with Phelan-McDermid Syndrome (PMS), KOALA, is currently enrolling participants. 18 sites have been activated. Study Type: Phase 3 Interventional Clinical Trial Age Range: children with PMS 3-12 years old Eligibility criteria apply Locations: Alabama, California, District of Columbia, Georgia, Illinois, Maryland, Massachusetts, Michigan, New York, Ohio, Texas, Virginia, and Toronto, Canada The Koala study is evaluating an investigational treatment in children with Phelan-McDermid syndrome. Each participant will be assigned by chance (like flipping a coin) to receive either the investigational treatment or a placebo. A placebo is an inactive treatment that looks like the investigational treatment but does not contain any active ingredient. This is a "double-blinded" study, which means neither you nor the study doctors would know which treatment your child is receiving. Participation in the study will last for a total of about 19 weeks. You will be expected to bring your child to 5 on-site clinic visits. You will also have 6 phone or video calls (your child does not need to attend these). Participants who complete the Koala study and meet eligibility requirements may have the option to participate in a separate 52-week open-label extension study. We’ll keep the community updated as new sites are added. To learn more about the Koala study, eligibility, and the participating in research, visit pms-study.com #NeurenPharmaceuticals #KoalaStudy #PhelanMcDermidSyndrome #PMSResearch

Today is Phelan-McDermid Syndrome Awareness Day! Every family impacted by Phelan-McDermid syndrome deserves to be seen, heard, and supported. Today, CureSHANK and PMSF are standing together to raise awareness for Phelan-McDermid syndrome. Join us as we raise awareness for the Phelan-McDermid syndrome community. #PMSAD #PhelanMcDermidSyndrome #PMSF #CureSHANK