Survey of muscle GSDs and COVID-19 issues.
IamGSD has launched a survey of people affected by muscle GSDs and how they are being affected by the COVID-19 pandemic.
Mendelian – reducing diagnostic delay in GSDs.
Mendelian, working to reduce diagnostic delay, and thus improve quality of life, in rare diseases including glycogen storage disease.
Valerion’s trial of therapy for Pompe stopped.
A trial of a new treatment for GSD2 has been halted. VAL-1221 was delivered intravenously in late-onset Pompe patients.
NICE seeks lay members for technology evaluation.
NICE are looking for lay members to join their highly specialised technologies evaluation committee.
Eurordis H-Care Survey of rare diseases.
AGSD-UK supports the Eurordis-led initiative of a survey of care in rare diseases across Europe. It takes just 10 minutes!
New information resources on gene therapy.
New information and videos – past, present and future of gene therapy from the American Society for Gene and Cell Therapy.