Information about the Phelan-McDermid Syndrome Foundation (PMSF)
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.
Over the past decade, more than 59,000 PHELAN LUCKY shirts have found homes in 33 countries across the globe. This has not only…
We are thrilled to invite you to join us in celebrating a momentous milestone - the 70th birthdays of two INCREDIBLE…
By Dr. Kate Still, April 2022 A large percentage (up to 80%) of people with a Phelan-McDermid syndrome diagnosis also have a…
by: Jen Randolph, PMSF Member & Donation Coordinator and Creator of Phelan-Lucky. THE WAIT IS OVER! 🍀 DATES: July 31st thru…
by: Ronni Blumenthal, Phelan-McDermid Syndrome Foundation CEO.Save the Date for the 2024 Phelan-McDermid Syndrome Foundation International Family ConferenceWe are so excited…
What is SHANK3? SHANK3 is a gene that is important for the development and function of the nervous system. Genes are…
PMSF announced a research grants program in January 2023 to provide funding to highly motivated scientists studying Phelan-McDermid syndrome. Our goal…
The new code is Q93.52 and will go into effect October 1, 2023. A years-long dream has been achieved for Phelan-McDermid…
