NNZ-2591 Phase 2 Publication
By Lauren Schmitt, PhDJanuary 12, 2026With Neuren's Phase 3 clinical trial for NNZ-2591 now open for enrollment, the recent publication of…
Why Sharing Clinical Trial Experiences Can Harm Research
By: Lauren Schmitt, PhD, Chief Science Officer, PMSFDate: December 22, 2025 How Families Can Help Protect the Path to New Treatments…
Neuren’s Phase 3 NNZ-2591 Trial for Phelan-McDermid Syndrome is Now Active!
Neuren’s Phase 3 NNZ-2591 Trial for Phelan-McDermid Syndrome is Now Active! by: Lauren Schmitt, PhDDate: December 15, 2025The Phelan-McDermid Syndrome Foundation is…
Class I vs. Class II Deletions: What’s the Difference?
In Phelan-McDermid syndrome, you are likely familiar with the terminology and differences between SHANK3 deletions, SHANK3 variations, and even ring chromosomes.…
PYC Therapeutics Webinar Takeaways
We are so grateful to PYC Therapeutics for their informative webinar on PYC-002, an RNA-based gene therapy, held on March 13,…
A Pilot Study Investigates a New Potential Treatment for Seizures in Phelan-McDermid Syndrome
This was written by PMSF to provide a summary of information in an accessible way for parents. PMSF does not speak…
Jaguar Gene Therapy Shares New Community Letter and FAQ with Latest Jag201 Clinical Study Details on ClinicalTrials.gov
Important Links:Updated Community Letter & FAQ from Jaguar Gene Therapy (11/5/24)Updated information is highlighted in blueJAG201 Clinical Study Record on ClinicalTrials.govJaguar's…
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting Phelan-McDermid syndrome
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting Phelan-McDermid syndrome
Protected: Jaguar Gene Therapy to Start a Clinical Trial in Phelan-McDermid syndrome
Jaguar Gene Therapy receives approval from FDA to conduct gene therapy clinical trials in Phelan-McDermid syndrome.
Jaguar Gene Therapy to Start a Clinical Trial in Phelan-McDermid syndrome
We have breaking news! Jaguar Gene Therapy has received approval from the FDA to conduct a gene therapy clinical trial in Phelan-McDermid syndrome. This is a major milestone for our community and for drug development in Phelan-McDermid syndrome! This will be the first trial aiming to target genetic underpinnings of Phelan-McDermid syndrome.