Dr. Weinstein, long-time champion of improved diagnosis and treatment for hepatic GSDs due to move to Passage Bio as VP.
Survey on rare disease patients’ experience of COVID-19 will enable EURORDIS to measure the effect on lives and inform decision-makers.
IamGSD has launched a survey of people affected by muscle GSDs and how they are being affected by the COVID-19 pandemic.
Mendelian, working to reduce diagnostic delay, and thus improve quality of life, in rare diseases including glycogen storage disease.
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.