Class I vs. Class II Deletions: What’s the Difference?
In Phelan-McDermid syndrome, you are likely familiar with the terminology and differences between SHANK3 deletions, SHANK3 variations, and even ring chromosomes.…
In Phelan-McDermid syndrome, you are likely familiar with the terminology and differences between SHANK3 deletions, SHANK3 variations, and even ring chromosomes.…
We are so grateful to PYC Therapeutics for their informative webinar on PYC-002, an RNA-based gene therapy, held on March 13,…
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting Phelan-McDermid syndrome
Jaguar Gene Therapy receives approval from FDA to conduct gene therapy clinical trials 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.
A large percentage (~75-80%) of people with a Phelan-McDermid syndrome diagnosis also have a diagnosis of autism spectrum disorder. This article…
What is SHANK3? SHANK3 is a gene that is important for the development and function of the nervous system. Genes are…
A research team at Northwestern University developed a new drug and tested it in a laboratory model of Phelan-McDermid syndrome.
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.
Do you wonder how a research publication affects your loved one or the field of Phelan-McDermid research?The Research Roundup is a…