Important links:

Newest FAQs Link 5/1/24 https://drive.google.com/file/d/1Wy6UmbJok6ZfxdB10c15YzQwj7f4dXvg/view?usp=sharing

Dear families,

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.

This treatment, called JAG201, has been in animal testing the past couple of years. PMSF has been in frequent contact with Jaguar to lower barriers to this clinical trial and amplify families’ voices.

Please note: this trial will be done on a very small number of people, dosed one at a time, with very specific criteria for inclusion. Participants will be adults with Phelan-McDermid syndrome. The first goal is to test safety. We will share more details as we have them.

For more information, check out the link above for a letter from Jaguar to the community with FAQs.

Kate Still, Ph.D.

PMSF Scientific Director