Dr. Weinstein, long-time champion of improved diagnosis and treatment for hepatic GSDs due to move to Passage Bio as VP.
The GSD3 get-together with the CATS at Cudworth Village Hall, on 14 March, has been postponed until later in the year.
Coming up on Saturday 14 March is another get-together for those with GSD3. Bring children, family and friends.
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.