Ultragenyx and Arcturus announce expansion of their research collaboration to develop gene therapies for rare diseases.
Dr Guiseppe Ronzitti, a group leader at Généthon, confirmed to speak on gene therapy developments for GSDs at our 2019 conference.
In May AGSD-UK facilitated a meeting of UK metabolic specialists to discuss guidelines for management of hepatic GSDs.
That is what member Niamh, who has GSD3, thought until she got a shock as she completed her GCSE Biology exam.
Saturday 30th March saw the CATS team celebrate their successful first year with a get-together with GSD3 friends.
Cori is a cat, with lots of kitten friends. He is on a mission to support children with GSD3.