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.
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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.
“First 100 Days”
Welcome to the “First 100 Days of your membership with the Phelan-McDermid Syndrome Foundation.
Caregiver Support Groups
We are thrilled to continue our support group program for Phelan-McDermid syndrome caregivers.
Winter Break Solutions
by: Carla D'Imperio, Director of Family Support.We gathered your ideas to help make the most of breaks from school and programs.We…
How are Phelan-McDermid Syndrome and Autism Related?
A large percentage (~75-80%) of people with a Phelan-McDermid syndrome diagnosis also have a diagnosis of autism spectrum disorder. This article…
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…
PMSF 2023 Grant Award Recipients
PMSF announced a research grants program in January 2023 to provide funding to highly motivated scientists studying Phelan-McDermid syndrome. Our goal…
Grief Support Group Registration
Registration is open for our first Phelan-McDermid Caregiver Support Group. We are thrilled to invite you to register for our new Caregiver Support Groups. These groups will be a time for Phelan-McDermid syndrome caregivers to share their experience in a welcoming and supportive group setting.