A Pilot Study Investigates a New Potential Treatment for Seizures in Phelan-McDermid Syndrome
This was written by PMSF to provide a summary of information in an accessible way for parents. PMSF does not speak…
Jaguar Gene Therapy Shares New Community Letter and FAQ with Latest Jag201 Clinical Study Details on ClinicalTrials.gov
Important Links:Updated Community Letter & FAQ from Jaguar Gene Therapy (11/5/24)Updated information is highlighted in blueJAG201 Clinical Study Record on ClinicalTrials.govJaguar's…
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.
PMSF engages parent advocate with Epilepsy Foundation
What was the event? PMS mom and PMSF rep Anna Williams participated at an annual Epilepsy Foundation Research Roundtable in Washington…
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 to co-fund Autism Science Foundation research grant
Dr. Sheng-Nan Qiao at Yale University applied to study the link between brain inflammation and regression (loss of skills) in PMS. The funded project focuses on approaches for reducing neuro-inflammation when modeling Phelan-McDermid syndrome in the laboratory.
What are antisense oligonucleotides (ASOs)?
We've been hearing about antisense oligonucleotides (ASOs) in the news as a potential therapeutic for PMS. But what are they and how do they work?