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…
Updates to Pharmacological Recommendations for the Management of Phelan-McDermid syndrome
We are excited to release updates to our Pharmacologic Recommendations, which were recently finalized by our Phelan-McDermid Syndrome Neuropsychiatric Consultation Group…
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.
SHANK3 in Phelan-McDermid syndrome
What is SHANK3? SHANK3 is a gene that is important for the development and function of the nervous system. Genes are…
New drug (JB2) has possible relevance to Phelan-McDermid syndrome
A research team at Northwestern University developed a new drug and tested it in a laboratory model of Phelan-McDermid syndrome.
Join us for a Patient-Focused Drug Development meeting with the FDA!
Save the date! November 8, 2022, 10 am - 3 pm ET (Please note this is election day in the U.S. …
Current Clinical Trial Recruitment Announcements in Phelan-McDermid syndrome
Note: The PMSF circulates information but does not promote individual clinical trials. Deciding to enroll in a clinical trial is a…