Support the call for new born screening.
New born screening has long been championed by AGSD-UK. Now you have an opportunity to support a larger national campaign.
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.
Dr Clive Tonks, ex-committee member of AGSD-UK.
We mark the passing of Dr Clive Tonks in his mid-eighties. He was a past trustee and committee member of AGSD-UK.
AGSD-UK helps fulfil football dreams for Ismail.
Ismail lives with Infantile Onset Pompe Disease. Aged 19, his abiding passion is football. He plays using an adapted wheelchair.
Netflix series “Diagnosis” refers to GSD.
A new series from Netflix, in which a doctor crowd-sources diagnoses for mysterious medical conditions, opens with a story mentioning GSD.
Gene therapy updates due at our 2019 conference
Dr Guiseppe Ronzitti, a group leader at Généthon, confirmed to speak on gene therapy developments for GSDs at our 2019 conference.