The first patients have been enrolled in the Genetx (Ultragenyx Pharmaceutical Inc) KIK-AS trial in the United States.
Fundraise for “Sleepless in September” by committing to a total of 8 hours of sleep between 6am Fri and 6:00 am on Sept 26 – 6am Sept 27th. https://sleeplessinseptember.gofundraise.com.au/
COVID-19 can be extra stressful for anyone with a family member with Angelman syndrome. We have compiled some information with expert advice that we will keep up to date as regularly as possible.
Two sites are open in Brisbane and Melbourne. Due to COVID-19 each site has evolving processes of scheduling screening visits, so it is important to be in direct contact with the study teams at the site closest to you.
“Receiving Fast Track designation for GTX-102 is an important recognition of our antisense oligonucleotide program and the promise it might offer to this patient population.” ~ Paula Evans, Chief Executive Officer, GeneTx.
Investigating the occurrence of food sensitivity and the relationship with gut microbiome in Angelman syndrome could lead to dietary intervention to help to reduce or alleviate symptoms
Two press releases were issued recently with one announcing an Exclusive Option Agreement between GeneTx Biotherapeutics, LLC (“GeneTx”) and Ultragenyx Pharmaceutical, Inc. (“Ultragenyx”), and the other announcing GeneTx’s lead candidate receiving Orphan Drug Designation (ODD) and Rare Pediatric Drug Designation (RPD). GeneTx would like to take this opportunity to provide additional information to our community about this very exciting news.
Felicity Williams joins the FAST board. Felicity’s combination of medical knowledge and position as a parent will be a asset to our team.
FAST Australia announces a new two-year Angelman syndrome research fellowship for Dr Emma Baker at the Murdoch Children’s Research Institute.