Jaguar Gene Therapy Will Target SHANK3 for Gene Therapy
JAG201 is designed to address mutation or deletion of the SHANK3 gene, a leading autism candidate gene, by delivering SHANK3 gene replacement therapy.
Posts about advances in Phelan-McDermid syndrome research
PMSF Research Roundup: December 2021
Do you wonder how a research publication affects your loved one or the field of Phelan-McDermid research?The Research Roundup is a…
Research Roundup – September 2021
Do you wonder how a research publication affects your loved one or the field of Phelan-McDermid research?The Research Roundup is a…
Research Roundup, August 2021
A monthly digest of publications regarding Phelan-McDermid syndrome. by Kate Still, Ph.D., PMSF Scientific Director A push towards Whole Genome Sequencing…
PMS Natural History Study is OPEN
By Katherine Still, Ph.DScientific Director Open enrollment for the Phelan-McDermid syndrome (PMS) Natural History Study The Natural History study is a…
International Survey for those living with Phelan-McDermid syndrome
by Diane Linnehan, Director of Operations One of our PMSF scientific advisors, Conny van Ravenswaaij, MD, Ph.D, a clinical geneticist from…
PMS Gastrointestinal Research – from Home
By Kathy Fiscus Operations Coordinator Research at HOME? What is a Smurfcake? The University of Miami and Atrium Health are conducting…
Kate Still PhD
PMSF Welcomes Scientific Director, Kate Still, PhDWe are very pleased to welcome Dr. Kate Still to our team as our full-time…
Dallman-lab
University of Miami Researcher Wins Approval for Phelan-McDermid GI Research StudyUniversity of Miami researcher Julia Dallman recently announced that her lab's…
Intranasal Oxytocin Study Results
Pilot Study Finds Intranasal Oxytocin Does Not Improve Autism Symptoms in Children with PMS-- letter submitted by Alex Kolevzon, MD Clinical…