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
July 9, 2024
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This page is your source for all of the latest news and blog posts about Phelan-McDermid syndrome (PMS).
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Endure4Eden: A 24-Hour Golf Challenge to Support the Phelan-McDermid Syndrome Foundation
We are excited to announce the upcoming Endure4Eden fundraiser, organized by Philip Robertson and his team, to support the Phelan-McDermid Syndrome Foundation. Taking place on
March 1, 2024
Protected: Jaguar Gene Therapy to Start a Clinical Trial in Phelan-McDermid syndrome
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January 31, 2024
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.
January 31, 2024
Current Open Research
*last updated on 3/4/2026* Profiling Responses and Immune Signatures in SHANK3-Mediated Neuropsychiatric Disease (PRISM) *This study is supported by the 2025 Shannon O’Boyle Memorial Neuropsychiatric
January 15, 2024
PHELAN LUCKY Sets Sights on $1 Million Goal
Over the past decade, more than 59,000 PHELAN LUCKY shirts have found homes in 33 countries across the globe. This has not only significantly increased awareness for
October 30, 2023
Katy & Sue’s Birthday
We are thrilled to invite you to join us in celebrating a momentous milestone – the 70th birthdays of two INCREDIBLE women who have made
September 26, 2023
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 explains why these two
September 25, 2023
Phelan Lucky is open until 8/22/23
by: Jen Randolph, PMSF Member & Donation Coordinator and Creator of Phelan-Lucky. THE WAIT IS OVER! 🍀 DATES: July 31st thru August 22nd The highly
July 31, 2023
2024 PMSF International Family Conference
by: Ronni Blumenthal, Phelan-McDermid Syndrome Foundation CEO. Save the Date for the 2024 Phelan-McDermid Syndrome Foundation International Family Conference We are so excited to announce
July 21, 2023
SHANK3 in Phelan-McDermid syndrome
SHANK3 is a gene that is important for the development and function of the nervous system. Genes are like instruction manuals in our bodies that
July 20, 2023
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 is to accelerate exciting
July 20, 2023