A trial of a new treatment for GSD2 has been halted. VAL-1221 was delivered intravenously in late-onset Pompe patients.
NICE are looking for lay members to join their highly specialised technologies evaluation committee.
AGSD-UK supports the Eurordis-led initiative of a survey of care in rare diseases across Europe. It takes just 10 minutes!
New information and videos – past, present and future of gene therapy from the American Society for Gene and Cell Therapy.
Development of services from Genomcs England continues apace, with the launch of a trial of whole genome sequencing for newborn babies.
The 5th International GSD Conference (IGSD2019) takes place in Porto Alegre, Brazil, from 14 to 16 November 2019.