Current Clinical Trial Recruitment Announcements in Phelan-McDermid syndrome
Note: The PMSF circulates information but does not promote individual clinical trials. Deciding to enroll in a clinical trial is a…
Applying for an ICD Code for Phelan-McDermid syndrome
https://youtu.be/fRDlkex2Kjg PMSF has applied to the CDC for an ICD Code for Phelan-McDermid syndrome. Our Scientific Director shares why this is…
CANDID Conference Resources
Topics this month in the PMSF Research Roundup: Assessment tool adapted to be more specific to PMS to be used in a clinical trial, Assessment tool adapted to be more specific to PMS to be used in a clinical trial, Assessment tool adapted to be more specific to PMS to be used in a clinical trial, Assessment tool adapted to be more specific to PMS to be used in a clinical trial.
New clinical trial results of Insulin-like Growth Factor-1 (IGF-1) in Phelan-McDermid syndrome
Results from a study testing the safety and feasibility of using growth hormone (GH) as a treatment in children with Phelan-McDermid syndrome has just been published.
Enrollment is currently open for Phase II of the Phelan-McDermid syndrome (PMS) Natural History Study
The Natural History Study is funded by NIH and the goal is to help medical professionals study PMS and how it progresses over time. This information is essential for developing clinical guidelines, identifying research priorities, and assessing the effectiveness of the clinical intervention.
A pilot study of Growth Hormone in children with Phelan-McDermid syndrome
Results from a study testing the safety and feasibility of using growth hormone (GH) as a treatment in children with Phelan-McDermid syndrome has just been published.
PMSF Scientific Advisory Committee Revisits Definition of Phelan-McDermid Syndrome
The Phelan-McDermid Syndrome Foundation Scientific Advisory Committee has proposed a new classification system for Phelan-McDermid syndrome in an important new publication.
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.
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…
Research Update
February 26, 2021Members of our research community have been asked to provide lay summaries that give a basic overview in clear…