Journal reviews progress on gene therapy for GSDs.
A paper in the journal “Human Molecular Genetics” reviews the development of gene therapy for glycogen storage diseases.
Dylan’s idea to help his friend leads to breakthroughs.
“So Chocolate Bar” – 6 years later and one boy’s idea to help his friend with GSD1b is leading to scientific breakthroughs.
Collaboration boosts prospect of GSD3 therapy.
Ultragenyx and Arcturus announce expansion of their research collaboration to develop gene therapies for rare diseases.
In case you missed these web site developments.
We have introduced a number of new features on the web site this year. Here is the low down.
Amy’s school Fun Run for her brother Eric.
Twin boys born with GSD1a and 8 year old big sister Amy organised a school Fun Run to help fund research.
Big leap forward in genetic diagnosis of rare diseases.
Under a plan by the NHS Genomic Medicine Service, genome sequencing is set to revolutionise the diagnosis of rare childhood conditions.