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
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.
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.
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…
New drug (JB2) has possible relevance to Phelan-McDermid syndrome
A research team at Northwestern University developed a new drug and tested it in a laboratory model of Phelan-McDermid syndrome.
PMSF Research Roundup: July 2022
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.
PMSF Research Roundup: May 2022
Do you wonder how a research publication affects your loved one or the field of Phelan-McDermid research?The Research Roundup is a…
PMSF Research Roundup: April 2022
Do you wonder how a research publication affects your loved one or the field of Phelan-McDermid research? The Research Roundup is…
PMSF Research Roundup: March 2022
Do you wonder how a research publication affects your loved one or the field of Phelan-McDermid research? The Research Roundup is…