Genetics Archives - Phelan-McDermid Syndrome Foundation

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.…

Epilepsy and Seizure Update from the Natural History Study

Seizures are a big concern for families with children with Phelan-McDermid syndrome, but it's not always clear when or why they…

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

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.