Phelan-McDermid Syndrome Study


The purpose of the study is to look at the safety, tolerability, and efficacy of NNZ-2591 in the treatment of children with Phelan-McDermid syndrome…

Info For Caregivers

You will be responsible for ensuring that the participant attends the study visits to meet with your study doctor and other study staff…


This clinical study is sponsored by Neuren Pharmaceuticals, a biopharmaceutical company headquartered in Melbourne, Australia…

Does someone you know have Phelan-McDermid syndrome?

They may be eligible to participate in a research study on a new medication for children and adolescents aged 3-12 years.

Phelan-McDermid syndrome is a genetically determined neurological disorder, characterized by intellectual disability, absence of speech or severe speech impediments, low muscle tone, developmental delays (including motor delays), symptoms of Autism Spectrum Disorder (ASD).

This is a spectrum disorder which means not all people display the same behaviors and symptoms.

People with Phelan-McDermid syndrome need continuous care and are unable to live independently.

There is currently no treatment for Phelan-McDermid syndrome.

Contact Us To Learn More

PMS Study Recruitment is now Closed.
Thank you to all the families who participated!