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.
Katy & Sue’s Birthday
We are thrilled to invite you to join us in celebrating a momentous milestone - the 70th birthdays of two INCREDIBLE…
How are Phelan-McDermid Syndrome and Autism Related?
By Dr. Kate Still, April 2022 A large percentage (up to 80%) of people with a Phelan-McDermid syndrome diagnosis also have a…
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.
What are antisense oligonucleotides (ASOs)?
We've been hearing about antisense oligonucleotides (ASOs) in the news as a potential therapeutic for PMS. But what are they and how do they work?
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…
Applying for an ICD Code for Phelan-McDermid syndrome
https://youtu.be/fRDlkex2Kjg PMSF has applied to the CDC for an ICD Code for Phelan-McDermid syndrome. Our Scientific Director shares why this is…